The New Frontiers of Gene Therapy and Gene Editing in Inflammatory Diseases

Hum Gene Ther. 2024 Apr;35(7-8):219-231. doi: 10.1089/hum.2023.210. Epub 2024 Mar 12.

Abstract

Inflammatory diseases are conditions characterized by abnormal and often excessive immune responses, leading to tissue and organ inflammation. The complexity of these disorders arises from the intricate interplay of genetic factors and immune responses, which challenges conventional therapeutic approaches. However, the field of genetic manipulation has sparked unprecedented optimism in addressing these complex disorders. This review aims to comprehensively explore the application of gene therapy and gene editing in the context of inflammatory diseases, offering solutions that range from correcting genetic defects to precise immune modulation. These therapies have exhibited remarkable potential in ameliorating symptoms, improving quality of life, and even achieving disease remission. As we delve into recent breakthroughs and therapeutic applications, we illustrate how these advancements offer novel and transformative solutions for conditions that have traditionally eluded conventional treatments. By examining successful case studies and preclinical research, we emphasize the favorable results and substantial transformative impacts that gene-based interventions have demonstrated in patients and animal models of inflammatory diseases such as chronic granulomatous disease, cryopyrin-associated syndromes, and adenosine deaminase 2 deficiency, as well as those of multifactorial origins such as arthropathies (osteoarthritis, rheumatoid arthritis) and inflammatory bowel disease. In conclusion, gene therapy and gene editing offer transformative opportunities to address the underlying causes of inflammatory diseases, ushering in a new era of precision medicine and providing hope for personalized, targeted treatments.

Keywords: CRISPR; chronic granulomatous disease; cryopyrin-associated syndromes; gene editing; gene therapy; hematopoietic stem progenitor cells; inflammatory bowel disease; inflammatory disorders; osteoarthritis; rheumatoid arthritis; viral vectors.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems
  • Gene Editing* / methods
  • Genetic Engineering
  • Genetic Therapy / methods
  • Humans
  • Quality of Life
  • Severe Combined Immunodeficiency*