Gene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies.
Hematol Oncol Clin North Am
; 36(4): 813-827, 2022 08.
Article
em En
| MEDLINE
| ID: mdl-35773051
ABSTRACT
Severe combined immune deficiency (SCID) causes profound deficiency in T cells and variable deficiencies in B and NK cells. Untreated, the condition is fatal within the first 2 years of life. HSCT has traditionally been the only curative approach; however, success rates are suboptimal in those lacking an HLA-matched donor and conditioning regimens can cause significant toxicity. Gene therapy was pioneered for adenosine deaminase (ADA-SCID) over 3 decades ago and has produced highly successful results. Encouraging data for X-SCID and preclinical work for Artemis-SCID and RAG1-SCID are paving the way for the therapy to become a viable curative treatment option.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Imunodeficiência Combinada Severa
Limite:
Humans
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article