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pkp2 gene therapy 在 New model yields promising gene therapy for heart condition 的相關結果

Scientists develop a credible heart cell model for ACM patients and observe a positive response in the condition using PKP2 gene therapy. ... <看更多>

pkp2 gene therapy 在 Plakophilin‐2 Gene Therapy Prevents Arrhythmogenic Right ... 的相關結果

We provide a novel mouse model that incorporates PKP2 patient genetics and serves as an ideal platform to evaluate therapies for ARVC. Early ... ... <看更多>

pkp2 gene therapy 在 Gene replacement therapy could help mend a broken heart ... 的相關結果

Gene replacement therapy could help mend a broken heart, study suggests ... One of these genes, PKP2, encodes a protein known as ... ... <看更多>

pkp2 gene therapy 在 Tenaya Therapeutics to Present Preclinical Data on TN-401 ... 的相關結果

Tenaya Therapeutics to Present Preclinical Data on TN-401 PKP2 Gene Therapy Program at Heart Rhythm 2022. Apr 28, 2022. ... <看更多>

pkp2 gene therapy 在 plakophilin-2 (pkp2) gene therapy using aav vector 的相關結果

Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 ... ... <看更多>

pkp2 gene therapy 在 Gene therapy composition and treatment of right ventricular ... 的相關結果

... dose of a gene therapy vector to cardiomyocytes of the human subject, wherein the gene therapy vector comprises a nucleic acid sequence encoding for PKP2. ... <看更多>

pkp2 gene therapy 在 Plakophilin-2 is required for transcription of genes ... - Nature 的相關結果

Plakophilin-2 (PKP2) is a component of the desmosome and known for its ... treatment of arrhythmias in patients with mutations in the gene ... ... <看更多>

pkp2 gene therapy 在 Mechanistic Insights and Therapeutic Approaches for ... 的相關結果

Late-stage AAV9 PKP2 treatment could similarly reassemble desmosomal proteins, ... Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a fatal genetic ... ... <看更多>

pkp2 gene therapy 在 Abstract 13599: LX2020, an Adeno Associated Viral-Based ... 的相關結果

To date, no effective treatment exists for ARVC. LX2020 is a novel AAV gene therapy that restores cardiac expression of human PKP2. ... <看更多>

pkp2 gene therapy 在 PKP2 gene: MedlinePlus Genetics 的相關結果

The PKP2 gene is found on chromosome 12. Related Health Topics. Genes and Gene Therapy · Genetic Disorders ... ... <看更多>

pkp2 gene therapy 在 Modeling reduced contractility and impaired desmosome ... 的相關結果

Loss-of-function mutations in PKP2, which encodes plakophilin-2, ... phenotypic analysis for proof of concept for gene replacement therapy. ... <看更多>

pkp2 gene therapy 在 Translation of New and Emerging Therapies for Genetic ... 的相關結果

Emerging treatments targets include gene therapy, myofilament function, ... Classic ARVC is caused primarily by variants in the genes PKP2, JUP, DSG2, ... ... <看更多>

pkp2 gene therapy 在 My heart will go on: patient-derived heart cells mimic disease ... 的相關結果

... platform for developing new treatments such as gene replacement therapy. ... One of these genes, PKP2, encodes a protein known as ... ... <看更多>

pkp2 gene therapy 在 tnya-ex991_6.htm 的相關結果

PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right Ventricular ... Mutations due to the PKP2 gene are the leading genetic cause of ARVC, ... ... <看更多>

pkp2 gene therapy 在 Lexeo Therapeutics | Gene Therapy Biotechnology | New York 的相關結果

Abstract highlights: A preclinical study of a novel mutated PKP2 knock-in mouse model that recapitulates all classic ARVC disease features found ... ... <看更多>

pkp2 gene therapy 在 Arrhythmogenic Cardiomyopathy: Exercise Pitfalls, Role of ... 的相關結果

Current therapies are aimed to prevent the worsening of disease phenotypes and ... and/or gene therapy—could very well improve cardiac function in ACM patients. ... <看更多>

pkp2 gene therapy 在 Tenaya Therapeutics on Twitter: "Data from our TN-401 PKP2 ... 的相關結果

Data from our TN-401 PKP2 gene therapy program for arrhythmogenic right ventricular cardiomyopathy were presented earlier this week at ... ... <看更多>

pkp2 gene therapy 在 LEXEO Therapeutics Announces Data Presentations at the ... 的相關結果

Study researchers hypothesized that PKP2 protein dose is a critical driver of ARVC, and that PKP2 gene therapy via AAV strategies can ... ... <看更多>

pkp2 gene therapy 在 siRNA, Anti-Human PKP2, 2'-F (SIRGT17403WQ-2F) 的相關結果

The product SIRGT17403WQ-2F is a type of small interfering RNA (siRNA) that targets PKP2 gene and regulates the expression of gene. ... <看更多>

pkp2 gene therapy 在 Usefulness of Genetic Study by Next-generation Sequencing ... 的相關結果

Bidirectional sequencing of the gene fragment (PKP2 in this case) of the ... therapy or as shocks in response to sustained ventricular arrhythmia. ... <看更多>

pkp2 gene therapy 在 Tenaya bags $75M from stock sale as it prepares to put a ... 的相關結果

... while TN-401 is another gene therapy targeted at genetic arrhythmogenic right ventricular cardiomyopathy caused by plakophilin 2 (PKP2) gene mutations. ... <看更多>

pkp2 gene therapy 在 Lexeo snaps up Stelios, bolstering pipeline with trio of rare ... 的相關結果

As Lexeo Therapeutics ushers two gene therapies toward the clinic, the company is adding ... one delivers the gene that codes for Plakophilin-2 (PKP2), ... ... <看更多>

pkp2 gene therapy 在 Cardiologists, Cardiothoracic Surgeons Available for Expert ... 的相關結果

... "Cardiac AAV: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Right Ventricular Remodeling, Improves Heart Function, ... ... <看更多>

pkp2 gene therapy 在 Stem Cell Reports - Article 的相關結果

tient with AC carrying a heterozygous frameshift PKP2 mutation. ... gene function may serve as an upstream therapy for the. ... <看更多>

pkp2 gene therapy 在 Kathy Ivey on LinkedIn: New preclinical updates from Tenaya ... 的相關結果

Leading Cardiac Regeneration and Gene Therapy Research and Programs at Tenaya ... “Cardiac AAV: PKP2 Gene Transfer Prevents Development of Arrhythmogenic ... ... <看更多>

pkp2 gene therapy 在 ARVD Newsletter Summer 2022.pdf - Johns Hopkins Medicine 的相關結果

genetic alteration. Many companies are interested in learning more about ARVC caused by PKP2 pathogenic variants and apply gene therapy techniques as a ... ... <看更多>

pkp2 gene therapy 在 Scaling New Heights in the Fight Against Heart Disease 的相關結果

Early clinical data for AAV gene therapy targeting cardiac muscle. (e.g. Danon Disease) ... PKP2. AAV. Genetic Arrhythmogenic RV. Cardiomyopathy (gARVC). ... <看更多>

pkp2 gene therapy 在 Plakophilin-2 is required for transcription of genes that control ... 的相關結果

potential treatment of arrhythmias in patients with mutations in. the gene coding for PKP2. Previous work has suggested that the structural ... ... <看更多>

pkp2 gene therapy 在 My heart will go on: Patient-derived heart cells mimic disease ... 的相關結果

One of these genes, PKP2, encodes a protein known as ... The findings from this study suggest that gene replacement therapy could be a ... ... <看更多>

pkp2 gene therapy 在 Anti-PKP2 (aa 125-139) polyclonal antibody (CPBT-56546GH) 的相關結果

This gene encodes a member of the arm-repeat (armadillo) and plakophilin gene families. Plakophilin proteins contain numerous armadillo repeats, ... ... <看更多>

pkp2 gene therapy 在 Tenaya Therapeutics Provides 2022 Business Updates 的相關結果

TN-401 - PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy (gARVC):. Tenaya has nominated TN-401 as a ... ... <看更多>

pkp2 gene therapy 在 Transcriptomic Coupling of PKP2 With Inflammatory and ... 的相關結果

Targeted anti-inflammatory therapy may be effective in ARVC. ... PKP2 mutations associate with most cases of gene-positive familial arrhythmogenic right ... ... <看更多>

pkp2 gene therapy 在 Novel frame-shift mutation in PKP2 associated with ... 的相關結果

Among family members mutation-specific genetic cascade screening has diagnostic, prognostic and therapeutic implications. Interpretation of test ... ... <看更多>

pkp2 gene therapy 在 Quantitative analysis of PKP2 and neighbouring genes in a ... 的相關結果

Aims. Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a disease mainly caused by desmosome gene mutations. The genetic culprit ... ... <看更多>

pkp2 gene therapy 在 Rocket Pharmaceuticals Inc Patent & Company Information 的相關結果

WO2022125489A1, Application, TREATMENT OF DANON DISEASE, June 16, 2022. US20220168446A1, Application, PLAKOPHILIN-2 (PKP2) GENE THERAPY USING AAV VECTOR ... ... <看更多>

pkp2 gene therapy 在 EN-510-02 - Heart Rhythm 的相關結果

Separate data show that reduced abundance of PKP2 leads ... (SupRep) KCNH2 gene therapy was created by cloning into a. ... <看更多>

pkp2 gene therapy 在 Tenaya Therapeutics Reports Third Quarter 2022 Financial ... 的相關結果

TN-401 – PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) o Tenaya plans to submit an IND ... ... <看更多>

pkp2 gene therapy 在 Homozygous truncating variant in PKP2 causes hypoplastic ... 的相關結果

Heterozygous variants in the PKP2 gene, encoding the desmosomal protein ... Surgical treatment, either staged palliation or cardiac transplantation, ... ... <看更多>

pkp2 gene therapy 在 Phenotypic Variability of a Pathogenic PKP2 Mutation in an ... 的相關結果

Pro105Leufs * 7) frameshift variant in the PKP2 gene. ... and sudden death of 13-year-old son, and therapy with beta-blocker and ACE inhibitors was initiated. ... <看更多>

pkp2 gene therapy 在 Arrhythmogenic Right Ventricular Cardiomyopathy: The Role ... 的相關結果

With the advancement of genetic testing and therapy, diseases such as ARVC may ... PKP2 is by far the most frequently involved gene in ARVC. ... <看更多>

pkp2 gene therapy 在 Arrhythmogenic Cardiomyopathy - Semantic Scholar 的相關結果

to usher in novel forms of therapy that will lead to improved care at ... Associated genes plakophilin-2 (PKP2), DSP, desmoglein-2 (DSG2),. ... <看更多>

pkp2 gene therapy 在 LEXEO Therapeutics Expands Cardiac Gene Therapy ... 的相關結果

LEXEO Therapeutics Expands Cardiac Gene Therapy Pipeline with ... 43 protein (Cx43) and the gene that encodes for Plakophilin-2 (PKP2), ... ... <看更多>

pkp2 gene therapy 在 New model yields promising gene therapy for heart condition | World ... 的相關結果

Scientists in Japan have developed a credible heart cell model for arrhythmogenic cardiomyopathy and observed a positive response in the condition using PKP2 ... ... <看更多>

pkp2 gene therapy 在 ESGCT Collaborative Virtual Congress 19–22 October 2021 ... 的相關結果

Retinal AAV gene therapy is emerging as the leading approach to treat different ... Cardiac AAV:PKP2 gene transfer prevents development of ... ... <看更多>

pkp2 gene therapy 在 Arrhythmogenic Cardiomyopathy: Exercise Pitfalls ... - MDPI 的相關結果

and functional benefits in ACM treatment and quality of life (QOL). ... coding gene plakophilin-2 (PKP2), critical for intracellular ... ... <看更多>

pkp2 gene therapy 在 gene therapy strategy to restore cardiac electrical and ... 的相關結果

2A ) and ARVC-PKP2mut ( FIG. ... 21A-B demonstrate cardiac connexin 43 gene therapy in neonates restores the cardiac mechanical junction ... ... <看更多>

pkp2 gene therapy 在 New frameshift mutation found in PKP2 gene in ... 的相關結果

a new frame shift mutation in the PKP2 gene through molecular genetic testing using Next ... antitachycardia pacing (ATP) therapy without ICD shocks. ... <看更多>

pkp2 gene therapy 在 Update on Genes Associated with Arrhythmogenic ... 的相關結果

There are some studies that have demonstrated the efficacy of ICD therapy in the ... Most of the ACM-linked pathogenic variants in the PKP2 gene have an ... ... <看更多>

pkp2 gene therapy 在 Cardiac and Aortic Panels Know&Manage - Medicover Genetics 的相關結果

Genetic information can improve clinical management by determining the right treatment and follow-up plan. It can predict a prognosis and therapy response ... ... <看更多>

pkp2 gene therapy 在 Matt Killeen, CSO at Renovacor Shares Insights on Pipeline ... 的相關結果

Matt spoke about the new research program for multiple genetic segments ... this AAV-based gene therapy program in parallel for PKP2, DSG2, ... ... <看更多>

pkp2 gene therapy 在 LEXEO Therapeutics to Present Clinical and Preclinical Data ... 的相關結果

Preclinical data from LX2020, an AAVrh10-based gene therapy candidate for the treatment of ARVC caused by mutations in the PKP2 gene; Interim ... ... <看更多>

pkp2 gene therapy 在 Medline ® Abstract for Reference 10 of 'Arrhythmogenic right ... 的相關結果

... right ventricular cardiomyopathy: Treatment and prognosis' ... CONCLUSIONS The introduction of the PKP2 R735X mutation into mice resulted in an ... ... <看更多>

pkp2 gene therapy 在 Arrhythmogenic cardiomyopathy: Identification of desmosomal ... 的相關結果

Leu797Gln, PKP2 p.Ser249Thr and p.E808fsX30). The results of the present investigation provided information on the search for modifier genes ... ... <看更多>

pkp2 gene therapy 在 Genetic testing for arrhythmogenic cardiomyopathy (R133) 的相關結果

PKP2, DSP, DSG2, DSC2, and JUP are desmosomal genes and are established causes of autosomal dominant ARVC. The majority of variants of significance are ... ... <看更多>

pkp2 gene therapy 在 Human genome - 臺北榮民總醫院的相關結果

OMIN) {[Of over 5,000 known genetic conditions, the molecular ... be treated with medication, dietary modification, or other therapies. ... <看更多>

pkp2 gene therapy 在 Juan A Bernal Rodriguez - CNIC 的相關結果

These viruses are generally used as tools in gene therapy and adapted to ... that PKP2 mutations alter actomyosin cytoskeleton and cardiac function, ... ... <看更多>

pkp2 gene therapy 在 Cardiomyopathy genetic testing - Royal Brompton Hospital 的相關結果

ARVD/C is typically inherited in an autosomal dominant manner [34] and most of the genes implicated in ARVC/D, such as PKP2, DSG2, DSP and DSC2 encode ... ... <看更多>

pkp2 gene therapy 在 ‪Jaclyn J Ho‬ - ‪Google 学术搜索‬ 的相關結果

Genetic regulators of a pluripotent adult stem cell system in planarians identified by ... Cardiac AAV: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, ... ... <看更多>

pkp2 gene therapy 在 Tenaya Therapeutics Completes 94,000 SQFT Genetic ... 的相關結果

Facility to Provide Clinical Supply of Lead Gene Therapy Programs TN-201 ... ventricular cardiomyopathy (ARVC) due to PKP2 gene mutations, ... ... <看更多>

pkp2 gene therapy 在 Genetic Testing - Medical Clinical Policy Bulletins - Aetna 的相關結果

Testing for sequence variants in the DSG2, DSP, and PKP2 genes in probands ... Genetic testing panels for the assessment and treatment of autism and other ... ... <看更多>

pkp2 gene therapy 在 Lab-grown heart cells 'mimic' disease in vitro 的相關結果

One of these genes, PKP2, encodes a protein known as ... rapid and convenient platform for developing gene-based therapies for this disease. ... <看更多>

pkp2 gene therapy 在 Recent advances in genetic testing and counseling for ... 的相關結果

Diagnostic, therapeutic, and prognostic values of genetic testing are most ... PKP2 gene mutations were most commonly found (54%) in a. ... <看更多>

pkp2 gene therapy 在 Lichao Liu (0000-0001-8259-281X) - ORCID 的相關結果

... patients with ARVD/C caused by heterozygous mutations in the PKP2 gene. ... Gene Therapy Against Doxorubicin-Induced Cardiotoxicity. ... <看更多>

pkp2 gene therapy 在 Genetic Testing for Inherited Cardiomyopathies and ... 的相關結果

Gene therapy could also be explored as an option, such as for Duchenne ... Variants in the PKP2 and TMEM43 genes have been found to be associated with ... ... <看更多>

pkp2 gene therapy 在 Cell models of arrhythmogenic cardiomyopathy: advances ... 的相關結果

Arrhythmogenic cardiomyopathy is a rare genetic disease that is ... and the uses of these models for future basic and therapeutic research. ... <看更多>

pkp2 gene therapy 在 Cardiac disease - Bulletin of Medical and Clinical Research 的相關結果

Cardiac disease: Current approaches to gene therapy. Md. Reyad-ul-ferdous a, b, ... Regulation of splicing of Several cardiac genes. DCM. ～3.7 613171. PKP2. ... <看更多>

pkp2 gene therapy 在 Renovacor, Inc. 2022 Current Report 8-K 的相關結果

... (AAV gene therapy) PKP2-associated ACM DSP-associated ACM IND Anticipated in 2H 2022 Commercial rights held by Renovacor. Genetic ACM ... ... <看更多>

pkp2 gene therapy 在 DiNAQOR – Bringing Gene Therapy to the Failing Heart 的相關結果

Spotlight. May 16, 2022. DiNAQOR Opens DiNAMIQS Subsidiary to Partner with Gene Therapy Companies Bringing New Treatments to ... ... <看更多>

pkp2 gene therapy 在 Indian Heart Journal - ScienceOpen 的相關結果

and 12 of plakophilin 2 (PKP2) gene in these patients. ... shock therapy was turned off after detailed discussion with the patient and his parents. ... <看更多>

pkp2 gene therapy 在 Genetic Testing for Hereditary Cardiomyopathies and ... - Cigna 的相關結果

Coverage Policies are not recommendations for treatment and should never be ... DSG2, DSP, JUP, PKP2, and TMEM43) ACM/ARVC genetic testing. ... <看更多>

pkp2 gene therapy 在 Gene therapy in sudden cardiac death: 的相關結果

Future therapy concepts of sudden cardiac death are currently the subject of ... mutations found in the desmosomal genes like JUP, DSP, PKP2, DSG2 and DSC2. ... <看更多>

pkp2 gene therapy 在 Actionable secondary findings in arrhythmogenic right ... 的相關結果

Cardiovascular Diagnosis and Therapy. ... No causal therapy is available for ARVC, so that the ... associated genes: PKP2, DSC2, DSG2, DSP and TMEM43. ... <看更多>

pkp2 gene therapy 在 Genetic predisposition study of heart failure and its ... 的相關結果

Plakophilin 2 (PKP2) gene mutations are prevalent [79]. ... The discovery of a harmful variant enhances therapeutic precision and reduces ... ... <看更多>

pkp2 gene therapy 在 A large familial pathogenic Plakophilin-2 gene (PKP2 ... 的相關結果

et al. Treatment of arrhythmogenic right ventricular cardiomyopathy/dysplasia: an International Task Force Consensus Statement. Circulation. ... <看更多>

pkp2 gene therapy 在 New Insight into the Variable Expression of Arrhythmogenic ... 的相關結果

Plakophilin 2 (PKP2) is the major disease-causing gene, accounting for 50–90% ... and for the development of future therapeutic strategies. ... <看更多>

pkp2 gene therapy 在 High proportion of genetic cases in patients with advanced ... 的相關結果

Of note, we identified a novel homozygous PKP2-gene mutation, ... and the lack of pharmacological evidence for preventive treatment. ... <看更多>

pkp2 gene therapy 在 A CASE REPORT: PKP2 GENE C.1592T>G VARIATION IN ... 的相關結果

Genetic testing of the plakophilin 2 (PKP2) gene was done by ... Treatment of Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia. ... <看更多>

pkp2 gene therapy 在 Links to the late-breaking studies at Heart Rhythm 2022 的相關結果

Cardiac AAV:PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Right Ventricular Remodeling, Improves Heart Function, ... ... <看更多>

pkp2 gene therapy 在 Insights Into Genetics and Pathophysiology of Arrhythmogenic ... 的相關結果

Arrhythmogenic cardiomyopathy (ACM) is a genetic disease characterized by ... The majority of PKP2 mutations are heterozygous nonsense, ... ... <看更多>

pkp2 gene therapy 在 Plakophilin-2 truncating variants impair cardiac contractility by ... 的相關結果

Truncating variants in the PKP2 gene represent 70 to 90% of all ... treatment strategies from other better studied disease such as DCM. ... <看更多>

pkp2 gene therapy 在 genes-11-00571.pdf - AIR Unimi 的相關結果

a Brugada-Syndrome-Associated PKP2 Mutation ... The patient was then discharged with sotalol therapy. The genetic. ... <看更多>

pkp2 gene therapy 在 Treatment modalities and prognosis of two arrhythmogenic ... 的相關結果

Keywords: Arrhythmogenic right ventricular cardiomyopathy, gene-phenotype, implantable cardiac devices implantation, PKP2 mutation, ... ... <看更多>

pkp2 gene therapy 在 Over 80 gene therapies in CVD pipeline with heart failure ... 的相關結果

By altering the genetic material of patients' cells, gene therapy has the potential to transform the treatment of highly medicated and costly ... ... <看更多>

pkp2 gene therapy 在 NHS to roll out life-saving gene therapy for rare disease ... 的相關結果

The revolutionary gene therapy treatment is known by its brand name Libmeldy and is used to treat metachromatic leukodystrophy (MLD), ... ... <看更多>

pkp2 gene therapy 在 Home | www.esgct 的相關結果

Human Gene Therapy ... Dr. Manfred Schmidt was a pioneer in the gene and cell therapy field, with more than 25 years experience in the sector. ... <看更多>

pkp2 gene therapy 在 Gene Therapy for Heart Failure 的相關結果

Gene Therapy for Heart Failure ... ACC.17 Scientific Session entitled "Evolving Therapies in Heart Failure: Gazing Into the Crystal Ball.". ... <看更多>

pkp2 gene therapy 在 StrideBio Announces Achievement of Significant Operational ... 的相關結果

... Supporting Transition of Novel Gene Therapy Pipeline to Clinical Stage ... of novel adeno-associated viral (AAV) based gene therapies, ... ... <看更多>

pkp2 gene therapy 在 Cardiac Genetic Mutation May Not Always Predict Heart Disease 的相關結果

“It's clear that these gene mutations have a real effect on one's heart, ... even on treatment, than patients with the condition who don't ... ... <看更多>

pkp2 gene therapy 在 New research moves novel gene therapy for heart failure ... 的相關結果

Knocking down the Hippo signaling pathway in pig cardiomyocytes after a heart attack, resulted in heart tissue renewal and improved ... ... <看更多>

pkp2 gene therapy 在 Breast Cancer Metastasis and Drug Resistance: Challenges and ... 的相關結果

Therapy will also depend on the cancer cell subtypes present in the patient, as well as the patient's menopausal status 8.4 Genetic Profile of Metastatic ... ... <看更多>

pkp2 gene therapy 在 Clinical and Molecular Aspects of Cardiomyopathies: On the ... 的相關結果

Table 1 Genetic background of arrhythmogenic cardiomyopathy MIM Entry Locus ... been identified in desmosomal genes, including DSP, plakophilin-2 (PKP2), ... ... <看更多>

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tenaya therapeutics

arvc

Scientists develop a credible heart cell model for ACM patients and observe a positive response in the condition using PKP2 gene therapy.

#2. Plakophilin‐2 Gene Therapy Prevents Arrhythmogenic Right ...

We provide a novel mouse model that incorporates PKP2 patient genetics and serves as an ideal platform to evaluate therapies for ARVC. Early ...

#3. Gene replacement therapy could help mend a broken heart ...

Gene replacement therapy could help mend a broken heart, study suggests ... One of these genes, PKP2, encodes a protein known as ...

#4. Tenaya Therapeutics to Present Preclinical Data on TN-401 ...

Tenaya Therapeutics to Present Preclinical Data on TN-401 PKP2 Gene Therapy Program at Heart Rhythm 2022. Apr 28, 2022.

#5. plakophilin-2 (pkp2) gene therapy using aav vector

Provided herein is a gene therapy for PKP2 (Plakophilin-2), e.g. using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 ...

#6. Gene therapy composition and treatment of right ventricular ...

... dose of a gene therapy vector to cardiomyocytes of the human subject, wherein the gene therapy vector comprises a nucleic acid sequence encoding for PKP2.

#7. Plakophilin-2 is required for transcription of genes ... - Nature

Plakophilin-2 (PKP2) is a component of the desmosome and known for its ... treatment of arrhythmias in patients with mutations in the gene ...

#8. Mechanistic Insights and Therapeutic Approaches for ...

Late-stage AAV9 PKP2 treatment could similarly reassemble desmosomal proteins, ... Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a fatal genetic ...

#9. Abstract 13599: LX2020, an Adeno Associated Viral-Based ...

To date, no effective treatment exists for ARVC. LX2020 is a novel AAV gene therapy that restores cardiac expression of human PKP2.

#10. PKP2 gene: MedlinePlus Genetics

The PKP2 gene is found on chromosome 12. Related Health Topics. Genes and Gene Therapy · Genetic Disorders ...

#11. Modeling reduced contractility and impaired desmosome ...

Loss-of-function mutations in PKP2, which encodes plakophilin-2, ... phenotypic analysis for proof of concept for gene replacement therapy.

#12. Translation of New and Emerging Therapies for Genetic ...

Emerging treatments targets include gene therapy, myofilament function, ... Classic ARVC is caused primarily by variants in the genes PKP2, JUP, DSG2, ...

#13. My heart will go on: patient-derived heart cells mimic disease ...

... platform for developing new treatments such as gene replacement therapy. ... One of these genes, PKP2, encodes a protein known as ...

#14. tnya-ex991_6.htm

PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right Ventricular ... Mutations due to the PKP2 gene are the leading genetic cause of ARVC, ...

#15. Lexeo Therapeutics | Gene Therapy Biotechnology | New York

Abstract highlights: A preclinical study of a novel mutated PKP2 knock-in mouse model that recapitulates all classic ARVC disease features found ...

#16. Arrhythmogenic Cardiomyopathy: Exercise Pitfalls, Role of ...

Current therapies are aimed to prevent the worsening of disease phenotypes and ... and/or gene therapy—could very well improve cardiac function in ACM patients.

#17. Tenaya Therapeutics on Twitter: "Data from our TN-401 PKP2 ...

Data from our TN-401 PKP2 gene therapy program for arrhythmogenic right ventricular cardiomyopathy were presented earlier this week at ...

#18. LEXEO Therapeutics Announces Data Presentations at the ...

Study researchers hypothesized that PKP2 protein dose is a critical driver of ARVC, and that PKP2 gene therapy via AAV strategies can ...

#19. siRNA, Anti-Human PKP2, 2'-F (SIRGT17403WQ-2F)

The product SIRGT17403WQ-2F is a type of small interfering RNA (siRNA) that targets PKP2 gene and regulates the expression of gene.

#20. Usefulness of Genetic Study by Next-generation Sequencing ...

Bidirectional sequencing of the gene fragment (PKP2 in this case) of the ... therapy or as shocks in response to sustained ventricular arrhythmia.

#21. Tenaya bags $75M from stock sale as it prepares to put a ...

... while TN-401 is another gene therapy targeted at genetic arrhythmogenic right ventricular cardiomyopathy caused by plakophilin 2 (PKP2) gene mutations.

#22. Lexeo snaps up Stelios, bolstering pipeline with trio of rare ...

As Lexeo Therapeutics ushers two gene therapies toward the clinic, the company is adding ... one delivers the gene that codes for Plakophilin-2 (PKP2), ...

#23. Cardiologists, Cardiothoracic Surgeons Available for Expert ...

... "Cardiac AAV: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Right Ventricular Remodeling, Improves Heart Function, ...

#24. Stem Cell Reports - Article

tient with AC carrying a heterozygous frameshift PKP2 mutation. ... gene function may serve as an upstream therapy for the.

#25. Kathy Ivey on LinkedIn: New preclinical updates from Tenaya ...

Leading Cardiac Regeneration and Gene Therapy Research and Programs at Tenaya ... “Cardiac AAV: PKP2 Gene Transfer Prevents Development of Arrhythmogenic ...

#26. ARVD Newsletter Summer 2022.pdf - Johns Hopkins Medicine

genetic alteration. Many companies are interested in learning more about ARVC caused by PKP2 pathogenic variants and apply gene therapy techniques as a ...

#27. Scaling New Heights in the Fight Against Heart Disease

Early clinical data for AAV gene therapy targeting cardiac muscle. (e.g. Danon Disease) ... PKP2. AAV. Genetic Arrhythmogenic RV. Cardiomyopathy (gARVC).

#28. Plakophilin-2 is required for transcription of genes that control ...

potential treatment of arrhythmias in patients with mutations in. the gene coding for PKP2. Previous work has suggested that the structural ...

#29. My heart will go on: Patient-derived heart cells mimic disease ...

One of these genes, PKP2, encodes a protein known as ... The findings from this study suggest that gene replacement therapy could be a ...

#30. Anti-PKP2 (aa 125-139) polyclonal antibody (CPBT-56546GH)

This gene encodes a member of the arm-repeat (armadillo) and plakophilin gene families. Plakophilin proteins contain numerous armadillo repeats, ...

#31. Tenaya Therapeutics Provides 2022 Business Updates

TN-401 - PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy (gARVC):. Tenaya has nominated TN-401 as a ...

#32. Transcriptomic Coupling of PKP2 With Inflammatory and ...

Targeted anti-inflammatory therapy may be effective in ARVC. ... PKP2 mutations associate with most cases of gene-positive familial arrhythmogenic right ...

#33. Novel frame-shift mutation in PKP2 associated with ...

Among family members mutation-specific genetic cascade screening has diagnostic, prognostic and therapeutic implications. Interpretation of test ...

#34. Quantitative analysis of PKP2 and neighbouring genes in a ...

Aims. Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a disease mainly caused by desmosome gene mutations. The genetic culprit ...

#35. Rocket Pharmaceuticals Inc Patent & Company Information

WO2022125489A1, Application, TREATMENT OF DANON DISEASE, June 16, 2022. US20220168446A1, Application, PLAKOPHILIN-2 (PKP2) GENE THERAPY USING AAV VECTOR ...

#36. EN-510-02 - Heart Rhythm

Separate data show that reduced abundance of PKP2 leads ... (SupRep) KCNH2 gene therapy was created by cloning into a.

#37. Tenaya Therapeutics Reports Third Quarter 2022 Financial ...

TN-401 – PKP2 Gene Therapy Program for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) o Tenaya plans to submit an IND ...

#38. Homozygous truncating variant in PKP2 causes hypoplastic ...

Heterozygous variants in the PKP2 gene, encoding the desmosomal protein ... Surgical treatment, either staged palliation or cardiac transplantation, ...

#39. Phenotypic Variability of a Pathogenic PKP2 Mutation in an ...

Pro105Leufs * 7) frameshift variant in the PKP2 gene. ... and sudden death of 13-year-old son, and therapy with beta-blocker and ACE inhibitors was initiated.

#40. Arrhythmogenic Right Ventricular Cardiomyopathy: The Role ...

With the advancement of genetic testing and therapy, diseases such as ARVC may ... PKP2 is by far the most frequently involved gene in ARVC.

#41. Arrhythmogenic Cardiomyopathy - Semantic Scholar

to usher in novel forms of therapy that will lead to improved care at ... Associated genes plakophilin-2 (PKP2), DSP, desmoglein-2 (DSG2),.

#42. LEXEO Therapeutics Expands Cardiac Gene Therapy ...

LEXEO Therapeutics Expands Cardiac Gene Therapy Pipeline with ... 43 protein (Cx43) and the gene that encodes for Plakophilin-2 (PKP2), ...

#43. New model yields promising gene therapy for heart condition | World ...

Scientists in Japan have developed a credible heart cell model for arrhythmogenic cardiomyopathy and observed a positive response in the condition using PKP2 ...

#44. ESGCT Collaborative Virtual Congress 19–22 October 2021 ...

Retinal AAV gene therapy is emerging as the leading approach to treat different ... Cardiac AAV:PKP2 gene transfer prevents development of ...

#45. Arrhythmogenic Cardiomyopathy: Exercise Pitfalls ... - MDPI

and functional benefits in ACM treatment and quality of life (QOL). ... coding gene plakophilin-2 (PKP2), critical for intracellular ...

#46. gene therapy strategy to restore cardiac electrical and ...

2A ) and ARVC-PKP2mut ( FIG. ... 21A-B demonstrate cardiac connexin 43 gene therapy in neonates restores the cardiac mechanical junction ...

#47. New frameshift mutation found in PKP2 gene in ...

a new frame shift mutation in the PKP2 gene through molecular genetic testing using Next ... antitachycardia pacing (ATP) therapy without ICD shocks.

#48. Update on Genes Associated with Arrhythmogenic ...

There are some studies that have demonstrated the efficacy of ICD therapy in the ... Most of the ACM-linked pathogenic variants in the PKP2 gene have an ...

#49. Cardiac and Aortic Panels Know&Manage - Medicover Genetics

Genetic information can improve clinical management by determining the right treatment and follow-up plan. It can predict a prognosis and therapy response ...

#50. Matt Killeen, CSO at Renovacor Shares Insights on Pipeline ...

Matt spoke about the new research program for multiple genetic segments ... this AAV-based gene therapy program in parallel for PKP2, DSG2, ...

#51. LEXEO Therapeutics to Present Clinical and Preclinical Data ...

Preclinical data from LX2020, an AAVrh10-based gene therapy candidate for the treatment of ARVC caused by mutations in the PKP2 gene; Interim ...

#52. Medline ® Abstract for Reference 10 of 'Arrhythmogenic right ...

... right ventricular cardiomyopathy: Treatment and prognosis' ... CONCLUSIONS The introduction of the PKP2 R735X mutation into mice resulted in an ...

#53. Arrhythmogenic cardiomyopathy: Identification of desmosomal ...

Leu797Gln, PKP2 p.Ser249Thr and p.E808fsX30). The results of the present investigation provided information on the search for modifier genes ...

#54. Genetic testing for arrhythmogenic cardiomyopathy (R133)

PKP2, DSP, DSG2, DSC2, and JUP are desmosomal genes and are established causes of autosomal dominant ARVC. The majority of variants of significance are ...

#55. Human genome - 臺北榮民總醫院

OMIN) {[Of over 5,000 known genetic conditions, the molecular ... be treated with medication, dietary modification, or other therapies.

#56. Juan A Bernal Rodriguez - CNIC

These viruses are generally used as tools in gene therapy and adapted to ... that PKP2 mutations alter actomyosin cytoskeleton and cardiac function, ...

#57. Cardiomyopathy genetic testing - Royal Brompton Hospital

ARVD/C is typically inherited in an autosomal dominant manner [34] and most of the genes implicated in ARVC/D, such as PKP2, DSG2, DSP and DSC2 encode ...

#58. ‪Jaclyn J Ho‬ - ‪Google 学术搜索‬

Genetic regulators of a pluripotent adult stem cell system in planarians identified by ... Cardiac AAV: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, ...

#59. Tenaya Therapeutics Completes 94,000 SQFT Genetic ...

Facility to Provide Clinical Supply of Lead Gene Therapy Programs TN-201 ... ventricular cardiomyopathy (ARVC) due to PKP2 gene mutations, ...

#60. Genetic Testing - Medical Clinical Policy Bulletins - Aetna

Testing for sequence variants in the DSG2, DSP, and PKP2 genes in probands ... Genetic testing panels for the assessment and treatment of autism and other ...

#61. Lab-grown heart cells 'mimic' disease in vitro

One of these genes, PKP2, encodes a protein known as ... rapid and convenient platform for developing gene-based therapies for this disease.

#62. Recent advances in genetic testing and counseling for ...

Diagnostic, therapeutic, and prognostic values of genetic testing are most ... PKP2 gene mutations were most commonly found (54%) in a.

#63. Lichao Liu (0000-0001-8259-281X) - ORCID

... patients with ARVD/C caused by heterozygous mutations in the PKP2 gene. ... Gene Therapy Against Doxorubicin-Induced Cardiotoxicity.

#64. Genetic Testing for Inherited Cardiomyopathies and ...

Gene therapy could also be explored as an option, such as for Duchenne ... Variants in the PKP2 and TMEM43 genes have been found to be associated with ...

#65. Cell models of arrhythmogenic cardiomyopathy: advances ...

Arrhythmogenic cardiomyopathy is a rare genetic disease that is ... and the uses of these models for future basic and therapeutic research.

#66. Cardiac disease - Bulletin of Medical and Clinical Research

Cardiac disease: Current approaches to gene therapy. Md. Reyad-ul-ferdous a, b, ... Regulation of splicing of Several cardiac genes. DCM. ～3.7 613171. PKP2.

#67. Renovacor, Inc. 2022 Current Report 8-K

... (AAV gene therapy) PKP2-associated ACM DSP-associated ACM IND Anticipated in 2H 2022 Commercial rights held by Renovacor. Genetic ACM ...

#68. DiNAQOR – Bringing Gene Therapy to the Failing Heart

Spotlight. May 16, 2022. DiNAQOR Opens DiNAMIQS Subsidiary to Partner with Gene Therapy Companies Bringing New Treatments to ...

#69. Indian Heart Journal - ScienceOpen

and 12 of plakophilin 2 (PKP2) gene in these patients. ... shock therapy was turned off after detailed discussion with the patient and his parents.

#70. Genetic Testing for Hereditary Cardiomyopathies and ... - Cigna

Coverage Policies are not recommendations for treatment and should never be ... DSG2, DSP, JUP, PKP2, and TMEM43) ACM/ARVC genetic testing.

#71. Gene therapy in sudden cardiac death:

Future therapy concepts of sudden cardiac death are currently the subject of ... mutations found in the desmosomal genes like JUP, DSP, PKP2, DSG2 and DSC2.

#72. Actionable secondary findings in arrhythmogenic right ...

Cardiovascular Diagnosis and Therapy. ... No causal therapy is available for ARVC, so that the ... associated genes: PKP2, DSC2, DSG2, DSP and TMEM43.

#73. Genetic predisposition study of heart failure and its ...

Plakophilin 2 (PKP2) gene mutations are prevalent [79]. ... The discovery of a harmful variant enhances therapeutic precision and reduces ...

#74. A large familial pathogenic Plakophilin-2 gene (PKP2 ...

et al. Treatment of arrhythmogenic right ventricular cardiomyopathy/dysplasia: an International Task Force Consensus Statement. Circulation.

#75. New Insight into the Variable Expression of Arrhythmogenic ...

Plakophilin 2 (PKP2) is the major disease-causing gene, accounting for 50–90% ... and for the development of future therapeutic strategies.

#76. High proportion of genetic cases in patients with advanced ...

Of note, we identified a novel homozygous PKP2-gene mutation, ... and the lack of pharmacological evidence for preventive treatment.

#77. A CASE REPORT: PKP2 GENE C.1592T>G VARIATION IN ...

Genetic testing of the plakophilin 2 (PKP2) gene was done by ... Treatment of Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia.

#78. Links to the late-breaking studies at Heart Rhythm 2022

Cardiac AAV:PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Right Ventricular Remodeling, Improves Heart Function, ...

#79. Insights Into Genetics and Pathophysiology of Arrhythmogenic ...

Arrhythmogenic cardiomyopathy (ACM) is a genetic disease characterized by ... The majority of PKP2 mutations are heterozygous nonsense, ...

#80. Plakophilin-2 truncating variants impair cardiac contractility by ...

Truncating variants in the PKP2 gene represent 70 to 90% of all ... treatment strategies from other better studied disease such as DCM.

#81. genes-11-00571.pdf - AIR Unimi

a Brugada-Syndrome-Associated PKP2 Mutation ... The patient was then discharged with sotalol therapy. The genetic.

#82. Treatment modalities and prognosis of two arrhythmogenic ...

Keywords: Arrhythmogenic right ventricular cardiomyopathy, gene-phenotype, implantable cardiac devices implantation, PKP2 mutation, ...

#83. Over 80 gene therapies in CVD pipeline with heart failure ...

By altering the genetic material of patients' cells, gene therapy has the potential to transform the treatment of highly medicated and costly ...

#84. NHS to roll out life-saving gene therapy for rare disease ...

The revolutionary gene therapy treatment is known by its brand name Libmeldy and is used to treat metachromatic leukodystrophy (MLD), ...

#85. Home | www.esgct

Human Gene Therapy ... Dr. Manfred Schmidt was a pioneer in the gene and cell therapy field, with more than 25 years experience in the sector.

#86. Gene Therapy for Heart Failure

Gene Therapy for Heart Failure ... ACC.17 Scientific Session entitled "Evolving Therapies in Heart Failure: Gazing Into the Crystal Ball.".

#87. StrideBio Announces Achievement of Significant Operational ...

... Supporting Transition of Novel Gene Therapy Pipeline to Clinical Stage ... of novel adeno-associated viral (AAV) based gene therapies, ...

#88. Cardiac Genetic Mutation May Not Always Predict Heart Disease

“It's clear that these gene mutations have a real effect on one's heart, ... even on treatment, than patients with the condition who don't ...

#89. New research moves novel gene therapy for heart failure ...

Knocking down the Hippo signaling pathway in pig cardiomyocytes after a heart attack, resulted in heart tissue renewal and improved ...

#90. Breast Cancer Metastasis and Drug Resistance: Challenges and ...

Therapy will also depend on the cancer cell subtypes present in the patient, as well as the patient's menopausal status 8.4 Genetic Profile of Metastatic ...

#91. Clinical and Molecular Aspects of Cardiomyopathies: On the ...

Table 1 Genetic background of arrhythmogenic cardiomyopathy MIM Entry Locus ... been identified in desmosomal genes, including DSP, plakophilin-2 (PKP2), ...