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Gene Therapy for Inborn Errors of Immunity.
Tiphaine Arlabosse1, Claire Booth2, Fabio Candotti3
1Pediatric Immuno-Rheumatology of Western Switzerland, Division of Pediatrics, Women-Mother-Child Department, Lausanne University Hospital, Lausanne, Switzerland.
The Journal of Allergy and Clinical Immunology. in Practice
|April 21, 2023
Summary
Gene therapy (GT) offers an alternative to stem cell transplants for severe inborn errors of immunity (IEIs). Advances in gene editing expand treatment options, though challenges remain for rare diseases.
Area of Science:
- Immunology
- Genetics
- Biotechnology
Background:
- Gene therapy (GT) emerged in the 1990s as an alternative to hematopoietic stem cell transplantation for severe inborn errors of immunity (IEIs).
- GT using autologous hematopoietic stem cells offers advantages like eliminating donor searches and graft-versus-host disease risks.
Conclusions:
- Gene therapy has shown promise for treating inborn errors of immunity, with ongoing advancements.
- Challenges persist, particularly concerning the safety of gene transfer tools and the economic viability for rare disease treatments.
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