Emerging therapies in genodermatoses

Clin Dermatol. 2020 Jul-Aug;38(4):462-466. doi: 10.1016/j.clindermatol.2020.03.006. Epub 2020 Mar 24.

Abstract

The human genome project yielded a compendium of genetic material that has allowed rapid advancement both in the technique of whole exome sequencing and also in the ability to identify single gene defects. The next generation of genetics has investigated how these genes interact in the development of disease, identifying pathways of illness and end organ tissue abnormal development. From the knowledge of single genes and pathways of genodermatosis development arises the opportunity to produce genetic therapies. This contribution reviews some of the exciting, emerging genetic therapies in genodermatoses.

Publication types

  • Review

MeSH terms

  • Administration, Topical
  • Anticholesteremic Agents / administration & dosage
  • Ectodermal Dysplasia 1, Anhidrotic / diagnosis
  • Ectodermal Dysplasia 1, Anhidrotic / genetics
  • Ectodermal Dysplasia 1, Anhidrotic / therapy
  • Epidermolysis Bullosa / diagnosis
  • Epidermolysis Bullosa / genetics
  • Epidermolysis Bullosa / therapy
  • Exome Sequencing
  • Gene Editing
  • Humans
  • Neurofibromatosis 1 / diagnosis
  • Neurofibromatosis 1 / genetics
  • Neurofibromatosis 1 / therapy
  • Signal Transduction / genetics
  • Skin Diseases, Genetic / diagnosis
  • Skin Diseases, Genetic / genetics
  • Skin Diseases, Genetic / therapy*
  • Ustekinumab / therapeutic use

Substances

  • Anticholesteremic Agents
  • Ustekinumab