Gene therapy for X-linked myotubular myopathy: the challenges
Gene therapy for X-linked myotubular myopathy: the challenges
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Publisher
England: Elsevier Limited
Journal title
Language
English
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Publication information
Publisher
England: Elsevier Limited
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Scope and Contents
Contents
[...]promising outcomes in relevant animal models can raise false expectations because the model might not fully reflect the human phenotype. [...]all stakeholders should view the transferability of such findings to the clinical setting with extreme caution. [...]the severity of hepatic involvement, and reports of myocarditis in ASPIRO and other ge...
Alternative Titles
Full title
Gene therapy for X-linked myotubular myopathy: the challenges
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TN_cdi_proquest_miscellaneous_2891759148
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2891759148
Other Identifiers
ISSN
1474-4422
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(23)00416-7
