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In vivo applications and toxicities of AAV-based gene therapies in rare diseases.

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Bibliographic Details
Title: In vivo applications and toxicities of AAV-based gene therapies in rare diseases.
Authors: Zhao, Qian1 (AUTHOR), Peng, Huifang1 (AUTHOR), Ma, Yujin1 (AUTHOR), Yuan, Huijun2 (AUTHOR) yuanhj301@wchscu.cn, Jiang, Hongwei1 (AUTHOR) jianghw@haust.edu.cn
Source: Orphanet Journal of Rare Diseases. 7/17/2025, Vol. 20 Issue 1, p1-14. 14p.
Database: Academic Search Ultimate
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Description
ISSN:17501172
DOI:10.1186/s13023-025-03893-z