Proof-of-concept for a gene replacement approach to CDKL5 deficiency disorder

Brain. 2020 Mar 1;143(3):716-718. doi: 10.1093/brain/awaa055.

Abstract

This scientific commentary refers to ‘Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder’, by Gao etal. (doi:10.1093/brain/awaa028).

Publication types

  • Comment

MeSH terms

  • Animals
  • Cyclins
  • Epileptic Syndromes*
  • Humans
  • Mice
  • Protein Serine-Threonine Kinases
  • Spasms, Infantile*

Substances

  • Cyclins
  • Protein Serine-Threonine Kinases
  • CDKL5 protein, human
  • CDKL5 protein, mouse

Supplementary concepts

  • CDKL5 deficiency disorder