AAV-directed muscular dystrophy gene therapy

Expert Opinion on Biological Therapy
Ying TangBing Wang

Abstract

Muscle-directed gene therapy for genetic muscle diseases can be performed by the recombinant adeno-associated viral (rAAV) vector delivery system to achieve long-term therapeutic gene transfer in all affected muscles. Recent progress in rAAV-vector-mediated muscle-directed gene transfer and associated techniques for the treatment of muscular dystrophies (MD). The review covers literature from the past 2 - 3 years. rAAV-directed muscular dystrophy gene therapy can be achieved by mini-dystrophin replacement and exon-skipping strategies. The additional strategies of enhancing muscle regeneration and reducing inflammation in the muscle micro-environment should be useful to optimize therapeutic efficacy. This review compares the merits and shortcomings of different administration methods, promoters and experimental animals that will guide the choice of the appropriate strategy for clinical trials. Restoration of muscle histopathology and function has been performed using rAAV systemic gene delivery. In addition, the combination of gene replacement and adjuvant therapies in the future may be beneficial with regard to improving muscle regeneration and decreasing myofiber necrosis. The challenges faced by large animal model studies and...Continue Reading

References

Sep 20, 1991·Cell·J M Ervasti, K P Campbell
Mar 1, 1990·International Journal of Cardiology·G NigroR J Bain
Feb 1, 1989·American Journal of Medical Genetics·A F MirandaM Rubin
Mar 1, 1982·Proceedings of the National Academy of Sciences of the United States of America·R J SamulskiN Muzyczka
Feb 1, 1983·Journal of Virology·A SrivastavaK I Berns
Feb 1, 1984·Proceedings of the National Academy of Sciences of the United States of America·G BulfieldK J Moore
Feb 1, 1995·The Journal of Cell Biology·C PasternakE L Elson
Apr 30, 1993·Biochemical and Biophysical Research Communications·L SenterG Salviati
Jul 1, 1996·The Journal of Cell Biology·J A RafaelJ S Chamberlain
Oct 1, 1996·Current Opinion in Cell Biology·M D Henry, K P Campbell
Nov 26, 1996·Proceedings of the National Academy of Sciences of the United States of America·P D KesslerB J Byrne
Apr 1, 1997·Human Molecular Genetics·V NigroG A Puca
May 23, 1998·Human Molecular Genetics·R H CrosbieK P Campbell
May 30, 1998·Proceedings of the National Academy of Sciences of the United States of America·M Imamura, E Ozawa
Sep 23, 1998·The Journal of Cell Biology·F DuclosK P Campbell
Nov 12, 1998·The American Journal of Pathology·V StraubK P Campbell
Dec 9, 1998·Proceedings of the National Academy of Sciences of the United States of America·G D ThomasR G Victor
Dec 29, 1998·The Journal of Cell Biology·Y M ChanL M Kunkel
Apr 16, 1999·Muscle & Nerve·P MelaciniC Angelini

❮ Previous
Next ❯

Citations

Mar 1, 2012·Cell and Tissue Research·Reiko ArakawaKayoko Saito
Dec 24, 2010·Molecular Therapy : the Journal of the American Society of Gene Therapy·Bo WuQi Long Lu
Jun 28, 2012·The Journal of Biological Chemistry·Bilal A AzakirMichael Sinnreich
Sep 24, 2010·Journal of Virology·Robert NgMavis Agbandje-McKenna
Apr 22, 2011·BMC Medical Genomics·Dwi U KemaladewiPeter A C 't Hoen
Nov 8, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Wenjing ZhangZefeng Wang
Oct 13, 2010·Annual Review of Pharmacology and Toxicology·Markus A Rüegg, David J Glass
Mar 13, 2014·Molecular Therapy : the Journal of the American Society of Gene Therapy·François MonjaretKarine Charton
Jan 25, 2011·Journal of Neuroscience Research·Jonathan J Magaña, Bulmaro Cisneros
May 28, 2011·Prenatal Diagnosis·Vedanta MehtaAnna L David
Mar 20, 2015·Drug Delivery and Translational Research·Elizabeth R LordenKam W Leong
Jun 7, 2014·Frontiers in Microbiology·Dwaipayan SenGiridhara R Jayandharan
Apr 14, 2016·Clinical Pharmacology and Therapeutics·Johnny HuardAiping Lu
Aug 16, 2012·Current Opinion in Neurology·Ingrid E C Verhaart, Annemieke Aartsma-Rus
May 6, 2014·Nature Genetics·Yuexiang WangJonathan A Fletcher

❮ Previous
Next ❯

Related Concepts

Related Feeds

Cell-Type-Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Cell-Type-Specific Viral Vectors (ASM)

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Cell-Type Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.