Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy

Hum Gene Ther. 2020 Jul;31(13-14):695-696. doi: 10.1089/hum.2020.182. Epub 2020 Jul 1.

Authors

James M Wilson¹, Terence R Flotte^{2

3}

Affiliations

¹ Gene Therapy Program, University of Pennsylvania, Perelman School of Medicine, Philadelphia, Pennsylvania, USA.
² Editor-in-Chief, Human Gene Therapy, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
³ Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA.

PMID: 32605399
DOI: 10.1089/hum.2020.182

No abstract available

Publication types

Editorial

MeSH terms

Genetic Therapy / methods*
Genetic Vectors / administration & dosage*
Humans
Myopathies, Structural, Congenital / genetics
Myopathies, Structural, Congenital / therapy*
Protein Tyrosine Phosphatases, Non-Receptor / genetics*

Substances

Protein Tyrosine Phosphatases, Non-Receptor