What a Clinician Needs to Know About Genome Editing: Status and Opportunities for Inborn Errors of Immunity

J Allergy Clin Immunol Pract. 2024 May;12(5):1139-1149. doi: 10.1016/j.jaip.2024.01.019. Epub 2024 Jan 19.


During the past 20 years, gene editing has emerged as a novel form of gene therapy. Since the publication of the first potentially therapeutic gene editing platform for genetic disorders, increasingly sophisticated editing technologies have been developed. As with viral vector-mediated gene addition, inborn errors of immunity are excellent candidate diseases for a corrective autologous hematopoietic stem cell gene editing strategy. Research on gene editing for inborn errors of immunity is still entirely preclinical, with no trials yet underway. However, with editing techniques maturing, scientists are investigating this novel form of gene therapy in context of an increasing number of inborn errors of immunity. Here, we present an overview of these studies and the recent progress moving these technologies closer to clinical benefit.

Keywords: Base editing; CRISPR/Cas; Gene editing; Inborn errors of immunity; Prime editing.

Publication types

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

MeSH terms

  • Agammaglobulinemia / genetics
  • Agammaglobulinemia / therapy
  • Animals
  • CRISPR-Cas Systems
  • Gene Editing* / methods
  • Genetic Therapy* / methods
  • Hematopoietic Stem Cell Transplantation
  • Humans
  • Severe Combined Immunodeficiency / genetics
  • Severe Combined Immunodeficiency / immunology
  • Severe Combined Immunodeficiency / therapy

Supplementary concepts

  • Severe combined immunodeficiency due to adenosine deaminase deficiency