Re: "Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy" by Wilson and Flotte
Hum Gene Ther
.
2020 Aug;31(15-16):787.
doi: 10.1089/hum.2020.217.
Authors
Perry B Shieh
1
,
Carsten G Bönnemann
2
,
Wolfgang Müller-Felber
3
,
Astrid Blaschek
3
,
James J Dowling
4
,
Nancy L Kuntz
5
,
Andreea M Seferian
6
Affiliations
1
University of California at Los Angeles School of Medicine, Los Angeles, California, USA.
2
National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA.
3
Klinikum der Universität München, Munich, Germany.
4
Hospital for Sick Children, Toronto, Ontario, Canada.
5
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, USA.
6
Institute of Myology, Pitié-Salpêtrière Hospital, Paris, France.
PMID:
32777938
PMCID:
PMC7462017
DOI:
10.1089/hum.2020.217
No abstract available
Publication types
Letter
Comment
MeSH terms
Genetic Therapy
Humans
Myopathies, Structural, Congenital* / genetics
Myopathies, Structural, Congenital* / therapy