Toward Combined Cell and Gene Therapy for Genodermatoses

Cold Spring Harb Perspect Biol. 2020 May 1;12(5):a035667. doi: 10.1101/cshperspect.a035667.


To date, more than 200 monogenic, often devastating, skin diseases have been described. Because of unmet medical needs, development of long-lasting and curative therapies has been consistently attempted, with the aim of correcting the underlying molecular defect. In this review, we will specifically address the few combined cell and gene therapy strategies that made it to the clinics. Based on these studies, what can be envisioned for the future is a patient-oriented strategy, built on the specific features of the individual in need. Most likely, a combination of different strategies, approaches, and advanced therapies will be required to reach the finish line at the end of the long and winding road hampering the achievement of definitive treatments for genodermatoses.

Publication types

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

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Epidermis / metabolism
  • Epidermolysis Bullosa / therapy
  • Epidermolysis Bullosa Dystrophica / therapy
  • Epidermolysis Bullosa, Junctional / therapy
  • Genes, Dominant
  • Genes, Recessive
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Humans
  • Lentivirus / genetics
  • Netherton Syndrome / therapy
  • Retroviridae / genetics
  • Simplexvirus
  • Skin / metabolism
  • Skin Diseases / genetics*
  • Skin Diseases / therapy*
  • Stem Cells / cytology