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Articles tagged with gene therapy

Progress in Gene Therapy for DMD: Slipping Full-Length Dystrophin Past the Immune System

The immune system is one of the biggest obstacles to gene therapy for Duchenne muscular dystrophy (DMD), but researchers have devised a new gene therapy method that can largely evade the body̵...

Tags: adenovirus, gene therapy.

Viral based Gene Therapy - A review by Dr Karl Bettelheim

Article submitted by Dr Karl Bettelheim published on the 3rd of June 2009 IntroductionIn my first review, I listed possible treatments under three headings and to remind readers...

Tags: AAV serotypes, Gene Therapy.

Crossing the Valley of Despair- A review by Dr Karl Bettelheim

Article submitted by Dr Karl Bettelheim published on the 3rd of June 2009Although there have been some very good studies published recently especially on the use of viruses to transfer human genes...

Tags: gene therapy, exonskipping, utrophin upregulation, immunodeficiency XSCID, EGFR gene, gefitinib.

Gene therapy in Muscular Dystrophy. - A review by Dr Karl Bettelheim

Article submitted by Dr Karl Bettelheim published on the 18th of July 2009There has recently appeared an excellent review (1) of current studies on gene therapy for muscular dystrophies. While poi...

Tags: Exonskipping, phosphorodiamidate morpholino oligomers, Gene therapy.

Transplantation of primary fibroblasts engineered to express a micro-dystrophin together with a tamoxifen-inducible form of the myogenic regulator MyoD.

Duchenne muscular dystrophy (DMD) is characterized in skeletal muscle by cycles of myofiber necrosis and regeneration leading to loss of muscle fibers and replacement with fibrotic connective and ...

Tags: gene therapy, Fibroblasts.

Successful transduction in neonatal dog, system wide with immunogenicity

Duchenne (DMD) and golden retriever (GRMD) muscular dystrophy are caused by genetic mutations in the dystrophin gene and afflict striated muscles. We investigated systemic gene delivery in 4-day-o...

Tags: AAV9, myofibers, gene therapy.

Microutrophin delivery through rAAV6 increases lifespan and improves muscle function in dystrophic dystrophin/utrophin-deficient mice

Duchenne muscular dystrophy (DMD), the most prevalent lethal genetic disorder in children, is caused by mutations in the 2.2-MB dystrophin gene. Absence of dystrophin and the dystrophin–glyco...

Tags: Microutrophin, gene therapy, dystrophin.

Restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx

Background— More than 90% of Duchenne muscular dystrophy (DMD) patients develop cardiomyopathy, and many die of cardiac failure. Despite tremendous progress in skeletal muscle gene therapy, f...

Tags: muscular dystrophy, genes, viruses, gene therapy, microdystrophin.

Transfer of the Full-Length Dystrophin-Coding Sequence into Muscle Cells by a Dual High-Capacity Hybrid Viral Vector with Site-Specific Integration Ability

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene, making it a potential target for gene therapy. There is, however, a scarcity of vectors that can accommodate the 14-kb DMD...

Tags: gene therapy.

ultrasound and microbubble mediated in vivo gene transfer.

Studies have shown that ultrasound, used either alone or in combination with microbubble contrast agents, can increase cell membrane permeability to plasmid DNA. Because ultrasound is a non-painfu...

Tags: gene transfer, gene therapy, dystrophic muscles, ultrasound.

Halofuginone and muscular dystrophy.

Institute of Animal Sciences, Volcani Center, Bet Dagan, Israel. pines@agri.huji.ac.il.Muscular dystrophies (MDs) include different inherited diseases that all result in progressive muscle degener...

Tags: Muscle, fibrosis, gene therapy, myoblast, Muscular dystrophies.

AAV-mediated gene therapy to the isolated limb in rhesus macaques.

Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital and Department of Pediatrics, The Ohio State University, Columbus, OH, USA.The development of a nonhuman pri...

Tags: gene therapy, limb, muscle, adenoassociated viral AAV.

Update on the Therapeutic approaches to muscular dystrophy

This is a summary of the article written by Aurelie Goyenvalle, Jane T Seto, Kay E Davies and Jeffrey Chamberlain, which was published by Oxford University Press on 5 April 2011.The article is a r...

Tags: exon skipping, gene therapy, Viral Vector, Dystrophin, rAAV, Antisense Oligonucleotide, PMO, PPMO, Duchenne, muscular dystrophy, Utrophin.