Novel Cross-Correction-Enabled Gene Therapy for CDKL5-Deficiency Disorder

Neurotherapeutics. 2022 Oct;19(6):1878-1882. doi: 10.1007/s13311-022-01314-8. Epub 2022 Oct 20.
No abstract available

Keywords: AAVPHP; CDKL5 deficiency; Cross-correction; Enzyme replacement therapy; Gene replacement therapy; TAT.

MeSH terms

  • Animals
  • Epileptic Syndromes* / genetics
  • Epileptic Syndromes* / therapy
  • Genetic Therapy
  • Humans
  • Mice
  • Mice, Knockout
  • Protein Serine-Threonine Kinases / genetics
  • Spasms, Infantile* / genetics
  • Spasms, Infantile* / therapy

Substances

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

Supplementary concepts

  • CDKL5 deficiency disorder