AAV Gene Therapy Prevents and Reverses Heart Failure in A ...

Gene Therapy With the N-Terminus of Junctophilin-2 Improves Heart ...

AAV Gene Therapy Prevents and Reverses Heart Failure in a Murine Knockout Model of Barth Syndrome · Medicine. Circulation research · 2020.

Gene therapy reverses advanced heart failure in animal study

Their method effectively and specifically delivered genes that promote proliferation to heart cells, generating new heart muscle. This approach ...

Cardiac AAV9-S100A1 Gene Therapy Rescues Post-Ischemic Heart ...

AAV vectors have proven effective and safe in small and large animal models of cardiovascular disease, 9, 10 and AAV serotype-9 (AAV9) vectors have been shown ...

ARV Cardiomyopathy Gene Therapy Gets Orphan Drug Designation

Safety evaluation of AAV:PKP2 in wild-type mice shows no adverse effects on cardiac function and no changes in tissues examined. Our preclinical ...

AAV8 gene therapy reverses cardiac pathology and prevents early ...

AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich's ataxia ... Authors: Joshua C. Chang; Molly R. Ryan ...

Phase 1 Clinical Trial of an Investigational Gene Therapy for ...

The goal of this initial clinical study is to assess safety and initial estimates of efficacy in treating heart disease. Anyone considering participating in a ...

AAV-based gene therapy prevents and halts the progression of ...

AAV-based gene therapy prevents and halts the progression of dilated cardiomyopathy in a mouse model of phosphoglucomutase 1 deficiency (PGM1-CDG). Bijina ...

Rescuing the Failing Heart by Targeted Gene Transfer

Gene therapy was initially applied in the clinical setting for inherited monogenic disorders. It is now apparent that gene therapy has broader potential that ...

Novel gene therapy works to reverse heart failure - Medical Xpress

Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested a powerful gene ...

Learn about Gene Therapy and How it Works

While there are several types of gene therapy, only AAV (adeno-associated virus) gene therapy is currently being investigated to treat cardiomyopathy and for ...

Our Science | Tenaya Therapeutics

DWORF Gene Therapy For Heart Failure (Discovery Stage) · Dwarf Open Reading Frame (DWORF) Gene Therapy Ameliorated Duchenne Muscular Dystrophy Cardiomyopathy in ...

Recent advances in cardiac gene therapy strategies targeting ...

Recombinant AAV (rAAV) vectors are especially advantageous for cardiac gene therapy because a number of AAV serotypes display natural tropism ...

Unique gene therapy prevents, reverses multiple sclerosis in animal ...

When the gene therapy was combined with immunotherapy using rapamycin — a drug used to coat heart stents and prevent organ transplant rejection ...

Reversing (heart) failure in Friedreich's ataxia

disease or its associated cardiomyopathy. Now, a team of French researchers has shown that i.v. frataxin gene therapy could prevent or even ...

Gene replacement therapy for Barth syndrome

In gene therapy for Barth syndrome, the goal is to deliver a new, working copy of tafazzin to prevent and reverse the impact of Barth syndrome on the heart and ...

Gene Therapy Reversed Heart Damage In Rats With Heart Failure

Long-term gene therapy resulted in improved cardiac function and reversed deterioration of the heart in rats with heart failure, according to a recent study.

Gene therapy vector-related myocarditis

Adeno-associated virus (AAV)-mediated gene replacement shows promise for targeted therapies in treatment of inherited cardiomyopathies and is the most used ...

A single combination gene therapy treats multiple age ... - PNAS

Indeed, an increased therapeutic effect was observed for AAV:sTGFβR2 combined with AAV:FGF21 or AAV:αKlotho in the renal and heart failure ...

Severe Friedreich's ataxia's heart defects reversed with gene therapy

Gene therapy safely reversed heart problems, lowered blood markers for heart muscle damage, and extended survival in a mouse model of severe Friedreich's ...

Potential of gene therapy as a treatment for heart failure - JCI

More recently AAV9 gene transfer of S100A1 in a preclinical model of ischemic cardiomyopathy induced dramatic improvements in contractile function, reinforcing ...