## 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 expression systems for gene transfer
✍ Scribed by Joseph E Rabinowitz; Jude Samulski
- Publisher
- Elsevier Science
- Year
- 1998
- Tongue
- English
- Weight
- 552 KB
- Volume
- 9
- Category
- Article
- ISSN
- 0958-1669
No coin nor oath required. For personal study only.
✦ Synopsis
In contrast to other gene delivery systems, adeno-associated virus vectors show long term gene expression without immune response or toxicity. New production methods have increased vector titers and eliminated adenovirus contamination, thereby facilitating effective in vivo use. These advancements will expedite additional animal model studies providing validation for use of this vector in human clinical trials.
📜 SIMILAR VOLUMES
## Abstract Adeno‐associated viral vectors (AAV) can direct long‐term gene expression in post‐mitotic cells. Previous studies have established that long‐term cardiac gene transfer results from intramuscular injection into the heart. Cardiac gene transfer after direct intracoronary delivery of AAV _
Several gene delivery vehicles are being developed for somatic gene therapy and each of these vectors has unique properties which makes them appropriate for different human disease applications. Recombinant adenoassociated viral (rAAV) vectors are proving themselves to be safe and ef®cacious for the
## Abstract ## Background Several genes are candidates for treating inner ear diseases. For clinical applications, minimally invasive approaches to the inner ear are desirable along with minimal side‐effects. ## Methods Adeno‐associated virus (AAV) was used as a vector into the guinea pig inner
## Abstract ## Background Gene transfer offers considerable potential for altering vessel wall physiology and intervention in vascular disease. Therefore, there is great interest in developing optimal strategies and vectors for efficient, targeted gene delivery into a vessel wall. ## Methods We
## 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