Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2010 May;30(2):237-48.
doi: 10.1016/j.iac.2010.02.002.

Gene therapy for primary immunodeficiencies

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

Gene therapy for primary immunodeficiencies

Alain Fischer et al. Immunol Allergy Clin North Am. 2010 May.

Erratum in

  • Immunol Allergy Clin North Am. 2011 May;31(2): I. Cavazanna-Calvo, M [corrected to Cavazzana-Calvo, M]

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.

PubMed Disclaimer

Republished in

  • Gene therapy for primary immunodeficiencies.
    Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Fischer A, et al. Hematol Oncol Clin North Am. 2011 Feb;25(1):89-100. doi: 10.1016/j.hoc.2010.11.007. Hematol Oncol Clin North Am. 2011. PMID: 21236392

MeSH terms

Substances