Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice-Reference-Cited by-同舟云学术

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Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice

Published:2023-07-27 Issue:1-2 Volume:31 Page:19-30
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Zhou Kaiyi,Yuan Meng,Sun Jiabao,Zhang Feixu,Zong Xiaoying,Li Zhanao,Tang Dingyue,Zhou Lichen,Zheng Jing,Xiao Xiao,Wu Xia^ORCID

Funder

National Natural Science Foundation of China

Shanghai Science and Technology Development Foundation

Department of Science and Technology of Sichuan Province

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.nature.com/articles/s41434-023-00411-3.pdf

Reference46 articles.

1. Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet. 2011;12:341–55.

2. Vandendriessche T, Thorrez L, Acosta-Sanchez A, Petrus I, Wang L, Ma L, et al. Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy. J Thromb Haemost. 2007;5:16–24.

3. Nathwani AC. Gene therapy for hemophilia. Hematol Am Soc Hematol Educ Program. 2019;2019:1–8.

4. Nagree MS, Scalia S, McKillop WM, Medin JA. An update on gene therapy for lysosomal storage disorders. Expert Opin Biol Ther. 2019;19:655–70.

5. Duan D. Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy. Mol Ther. 2018;26:2337–56.

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