## Abstract Although the remarkable versatility and efficacy of recombinant adeno‐associated virus 2 (AAV2) vectors in transducing a wide variety of cells and tissues in vitro, and in numerous pre‐clinical animal models of human diseases in vivo, have been well established, the published literature
Adeno-Associated Virus —Mediated Gene Transfer for Hemophilia B
✍ Scribed by Katherine A. High
- Book ID
- 105675597
- Publisher
- Carden Jennings Publishing
- Year
- 2002
- Tongue
- English
- Weight
- 111 KB
- Volume
- 76
- Category
- Article
- ISSN
- 0925-5710
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## Abstract ## Background Photoreceptor (PR) and retinal pigment epithelium (RPE) are the principal cell targets in retinal gene therapy. Recombinant adeno‐associated virus (rAAV) has emerged as a very promising vector for gene therapy in hereditary retinal diseases. Gene transfer at different sta