OP018: ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findings-Reference-Cited by-同舟云学术

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OP018: ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findings

Published:2022-03 Issue:3 Volume:24 Page:S350
ISSN:1098-3600
Container-title:Genetics in Medicine
language:en
Short-container-title:Genetics in Medicine

Author:

Shieh Perry,Kuntz Nancy,Dowling James,Müller-Felber Wolfgang,Bönnemann Carsten,Foley Dimah,Seferian Andreea,Servais Laurent,Lawlor Michael,Noursalehi Mojtaba,Prasad Suyash,Rico Salvador,Miller Weston

Publisher

Elsevier BV

Subject

Genetics (clinical)

Cited by 7 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Current developments of gene therapy in human diseases;MedComm;2024-08-16

2. Comparative in vivo characterization of newly discovered myotropic adeno-associated vectors;Skeletal Muscle;2024-05-03

3. Molecular mechanisms and therapeutic strategies for neuromuscular diseases;Cellular and Molecular Life Sciences;2024-04-28

4. Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques;Molecular Therapy - Methods & Clinical Development;2024-03

5. Comparative in vivo characterization of newly discovered myotropic adeno-associated vectors;2023-09-01

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