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- Title
Breaking the barriers to global gene delivery.
- Authors
Weitzman, Matthew D; Thistlethwaite, Patricia A
- Abstract
The article presents information on various technology used in global gene delivery. For both scientist and physician, the biggest challenge to the use of viral vectors for gene delivery is how to introduce and express the therapeutic gene in a sufficient number of cells to achieve clinical efficacy. Optimal treatment of muscular dystrophy and other skeletal-muscle degenerative diseases requires gene delivery to major muscle groups throughout the body. To achieve this goal, several approaches using adeno-associated virus, AAV have been taken. Vectors for gene therapy should be tailored to the disease being treated. Molecular treatments of cardiac disease must be highly focused to one organ, with minimal extraorgan tropism and cytotoxicity.
- Subjects
BIOTECHNOLOGY; ADENOVIRUS diseases; GENE therapy; CELL-mediated cytotoxicity; GENETIC engineering; PHYSICIANS
- Publication
Nature Biotechnology, 2005, Vol 23, Issue 3, p305
- ISSN
1087-0156
- Publication type
Article
- DOI
10.1038/nbt0305-305