Gene Therapy Strategies for Duchenne Muscular Dystrophy Utilizing Recombinant Adeno-associated Virus Vectors
Open Access
- 1 February 2006
- journal article
- review article
- Published by Elsevier in Molecular Therapy
- Vol. 13 (2) , 241-249
- https://doi.org/10.1016/j.ymthe.2005.11.001
Abstract
No abstract availableKeywords
This publication has 53 references indexed in Scilit:
- Adeno-associated virus and the development of adeno-associated virus vectors: a historical perspectiveMolecular Therapy, 2004
- Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1Blood, 2004
- From Virus Evolution to Vector Revolution: Use of Naturally Occurring Serotypes of Adeno-associated Virus (AAV) as Novel Vectors for Human Gene TherapyCurrent Gene Therapy, 2003
- AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia BBlood, 2003
- Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene productGene Therapy, 2002
- Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophyNature Medicine, 2002
- The dystrophin–glycoprotein complex, cellular signaling, and the regulation of cell survival in the muscular dystrophiesMuscle & Nerve, 2001
- Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeuticsNature Medicine, 2001
- Route of Administration Determines Induction of T-Cell-Independent Humoral Responses to Adeno-Associated Virus VectorsMolecular Therapy, 2000
- The complete sequence of dystrophin predicts a rod-shaped cytoskeletal proteinCell, 1988