Lipid Nanoparticles Efficiently Deliver the Base Editor ABE8e for COL7A1 Correction in Dystrophic Epidermolysis Bullosa Fibroblasts In Vitro

J Invest Dermatol. 2024 Oct;144(10):2314-2317.e3. doi: 10.1016/j.jid.2024.03.027. Epub 2024 Apr 5.
No abstract available

Keywords: Epidermolysis bullosa; Gene editing; Gene therapy; Lipid nanoparticles.

Publication types

  • Letter

MeSH terms

  • Cells, Cultured
  • Collagen Type VII* / genetics
  • Collagen Type VII* / metabolism
  • Epidermolysis Bullosa Dystrophica* / genetics
  • Epidermolysis Bullosa Dystrophica* / metabolism
  • Epidermolysis Bullosa Dystrophica* / pathology
  • Epidermolysis Bullosa Dystrophica* / therapy
  • Fibroblasts* / metabolism
  • Genetic Therapy / methods
  • Humans
  • Lipids / chemistry
  • Liposomes
  • Nanoparticles*

Substances

  • Collagen Type VII
  • COL7A1 protein, human
  • Lipid Nanoparticles
  • Liposomes
  • Lipids