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 REPLACEMENT THERAPY DEVELOPMENT FOR BARTH ...

Why is Barth syndrome an ideal target for a gene therapy? Barth syndrome happens when there is something wrong with the tafazzin gene.

AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and ...

Barth syndrome (BTHS) is a rare mitochondrial disease that affects heart and skeletal muscle and has no curative treatment. It is caused by recessive ...

Gene therapy for heart, skeletal muscle disorder shows promise in ...

Barth syndrome stalks young patients, weakening heart and skeletal muscles, stunting growth and shortening their lives.

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

Currently, there is no targeted treatment for Barth syndrome. Lack of a proper genetic animal model that recapitulates the features of Barth ...

Gene therapy for Barth syndrome | Harvard Stem Cell Institute (HSCI)

HSCI researchers have successfully tested a gene therapy in mice for Barth syndrome, a rare disease that can cause life-threatening heart ...

Gene therapy reverses Barth syndrome in mice

Gene therapy reverses heart failure in mouse model of Barth syndrome · Barth syndrome is a rare metabolic disease caused by mutation of a gene ...

Current and future treatment approaches for Barth syndrome

Barth syndrome is an excellent candidate for AAV-based gene therapy. TAFAZZIN mutations in Barth syndrome cause loss-of-function, suggesting ...

AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic ...

Barth syndrome (BTHS) is a rare mitochondrial disease that causes severe cardiomyopathy and has no disease-modifying therapy.

Gene therapy reverses heart failure in mouse model of Barth ...

Barth syndrome is a rare genetic disease in boys that can cause life-threatening heart failure and also weakens the skeletal muscles and the ...

TAZ gene therapy reverses Barth syndrome in mice

Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.

"AAV-mediated TAZ gene replacement restores mitochondrial and ...

Todd; and Pacak, Christina A., "AAV-mediated TAZ gene replacement restores mitochondrial and cardioskeletal function in Barth syndrome." Human Gene Therapy.

Barth Syndrome Cardiomyopathy: An Update - MDPI

Barth syndrome (BTHS) is an X-linked mitochondrial lipid disorder caused by mutations in the TAFAZZIN (TAZ) gene, which encodes a mitochondrial acyltransferase ...

Potential Therapies for BTHS - Barth Syndrome Foundation

Gene therapy vector optimization and testing for Barth syndrome. Christina Pacak, PhD — University of Florida, Gainesville, FL

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

Rationale: Barth syndrome (BTHS) is an X-linked cardiac and skeletal myopathy caused by mutation of the gene Tafazzin (TAZ). Currently there is no targeted ...

Gene Therapy Reverses Heart Failure in Animal Model of Barth ...

Boston Children's Hospital researchers used an investigational gene therapy to treat heart failure in a mouse model of Barth syndrome.

Current and Future Treatment Approaches for Barth Syndrome

... straightforward treatment method. Currently, two approaches aiming to restore TAZ in BTHS are under investigation: gene therapy based on adeno-associated ...

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

AAV Gene Therapy Prevents and Reverses Heart Failure in a Murine Knockout Model of Barth Syndrome.

Barth syndrome: mechanisms and management | TACG

No variants in other genes have been reported to cause Barth syndrome. Rarely, Barth syndrome may be due to microdeletions of the X-chromosome.

Tafazzin gene therapy created to treat Barth syndrome - BioWorld

TAZ gene replacement therefore appeared to have the potential to improve important characteristics of Barth syndrome and warrants evaluation for ...