Redirecting AAV vectors to extrahepatic tissues.
Publication
, Journal Article
Asokan, A; Shen, S
Published in: Mol Ther
December 6, 2023
Recombinant adeno-associated viral (AAV) vectors are the current benchmark for systemic delivery of gene therapies to multiple organs in vivo. Despite clinical successes, safe and effective gene delivery to extrahepatic tissues has proven challenging due to dose limiting toxicity arising from high liver uptake of AAV vectors. Deeper understanding of AAV structure, receptor biology, and pharmacology has enabled the design and engineering of liver-de-targeted capsids ushering in several new vector candidates. This next generation of AAVs offers significant promise for extrahepatic gene delivery to cardiovascular, musculoskeletal, and neurological tissues with improved safety profiles.
Duke Scholars
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Published In
Mol Ther
DOI
EISSN
1525-0024
Publication Date
December 6, 2023
Volume
31
Issue
12
Start / End Page
3371 / 3375
Location
United States
Related Subject Headings
- Liver
- Genetic Vectors
- Genetic Therapy
- Gene Transfer Techniques
- Dependovirus
- Capsid
- Biotechnology
- 3206 Medical biotechnology
- 3202 Clinical sciences
- 3105 Genetics
Citation
APA
Chicago
ICMJE
MLA
NLM
Asokan, A., & Shen, S. (2023). Redirecting AAV vectors to extrahepatic tissues. Mol Ther, 31(12), 3371–3375. https://doi.org/10.1016/j.ymthe.2023.10.005
Asokan, Aravind, and Shen Shen. “Redirecting AAV vectors to extrahepatic tissues.” Mol Ther 31, no. 12 (December 6, 2023): 3371–75. https://doi.org/10.1016/j.ymthe.2023.10.005.
Asokan A, Shen S. Redirecting AAV vectors to extrahepatic tissues. Mol Ther. 2023 Dec 6;31(12):3371–5.
Asokan, Aravind, and Shen Shen. “Redirecting AAV vectors to extrahepatic tissues.” Mol Ther, vol. 31, no. 12, Dec. 2023, pp. 3371–75. Pubmed, doi:10.1016/j.ymthe.2023.10.005.
Asokan A, Shen S. Redirecting AAV vectors to extrahepatic tissues. Mol Ther. 2023 Dec 6;31(12):3371–3375.
Published In
Mol Ther
DOI
EISSN
1525-0024
Publication Date
December 6, 2023
Volume
31
Issue
12
Start / End Page
3371 / 3375
Location
United States
Related Subject Headings
- Liver
- Genetic Vectors
- Genetic Therapy
- Gene Transfer Techniques
- Dependovirus
- Capsid
- Biotechnology
- 3206 Medical biotechnology
- 3202 Clinical sciences
- 3105 Genetics