High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy-Reference-Cited by-同舟云学术

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High-throughput transcriptome analyses from ASPIRO, a phase 1/2/3 study of gene replacement therapy for X-linked myotubular myopathy

Published:2023-10 Issue:10 Volume:110 Page:1648-1660
ISSN:0002-9297
Container-title:The American Journal of Human Genetics
language:en
Short-container-title:The American Journal of Human Genetics

Author:

Andreoletti Gaia^ORCID,Romano Oriana,Chou Hsin-Jung,Sefid-Dashti Mahjoubeh J.,Grilli Andrea,Chen Clarice,Lakshman Neema,Purushothaman Pravin,Varfaj Fatbardha,Mavilio Fulvio,Bicciato Silvio,Urbinati Fabrizia

Publisher

Elsevier BV

Subject

Genetics (clinical),Genetics

Reference48 articles.

1. Genomic organization of the MTM1 gene implicated in X-linked myotubular myopathy;Laporte;Eur. J. Hum. Genet.,1998

2. Costs and health resource use in patients with X-linked myotubular myopathy: insights from US commercial claims;Sacks;J. Manag. Care Spec. Pharm.,2021

3. The myotubularin family: from genetic disease to phosphoinositide metabolism;Laporte;Trends Genet.,2001

4. X-Linked Myotubular Myopathy;Dowling,1993

5. A natural history study of X-linked myotubular myopathy;Amburgey;Neurology,2017

Cited by 1 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. Congenital myopathies: pathophysiological mechanisms and promising therapies;Journal of Translational Medicine;2024-09-02

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