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. 2011 Feb;25(1):89-100.
doi: 10.1016/j.hoc.2010.11.007.

Gene therapy for primary immunodeficiencies

Alain Fischer  1 , S Hacein-Bey-Abina, M Cavazzana-Calvo
Affiliations

Gene therapy for primary immunodeficiencies

Alain Fischer et al. Hematol Oncol Clin North Am. 2011 Feb.

Abstract

The concept of gene therapy emerged as a way of correcting monogenic inherited diseases by introducing a normal copy of the mutated gene into at least some of the patients' cells. Although this concept has turned out to be quite complicated to implement, it is in the field of primary immunodeficiencies (PIDs) that proof of feasibility has been undoubtedly achieved. There is now a strong rationale in support of gene therapy for at least some PIDs, as discussed in this article.

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Republished from

  • Gene therapy for primary immunodeficiencies.
    Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Fischer A, et al. Immunol Allergy Clin North Am. 2010 May;30(2):237-48. doi: 10.1016/j.iac.2010.02.002. Immunol Allergy Clin North Am. 2010. PMID: 20493399 Review.

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