One transgene, two myopathies: an MTM1 'cross gene therapy' for BIN1 deficiency?

Brain. 2023 Oct 3;146(10):3966-3968. doi: 10.1093/brain/awad310.
No abstract available

Publication types

  • Research Support, Non-U.S. Gov't
  • Comment

MeSH terms

  • Adaptor Proteins, Signal Transducing / genetics
  • Humans
  • Myopathies, Structural, Congenital* / genetics
  • Myopathies, Structural, Congenital* / therapy
  • Nuclear Proteins / genetics
  • Tumor Suppressor Proteins / genetics

Substances

  • BIN1 protein, human
  • Nuclear Proteins
  • Tumor Suppressor Proteins
  • Adaptor Proteins, Signal Transducing