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Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID).
Methods Enzymol. 2012; 507:15-27.ME

Abstract

Severe combined immunodeficiencies (SCIDs) appear as optimal disease targets to challenge potential efficacy of gene therapy. Ex vivo, retrovirally mediated gene transfer into hematopoietic progenitor cells has been shown to provide sustained correction of two forms of SCID, that is, SCID-X1 and adenosine deaminase deficiencies. In the former case, however, genotoxicity was observed in a minority of patients as a consequence of retroviral integration into proto-oncogenes loci and transactivation. Design of vectors in which the enhancer element of retroviral LTR has been deleted and an internal promoter added (self-inactivated vectors) could provide both safe and efficient gene transfer as being presently tested.

Authors+Show Affiliations

Descartes University of Paris, Paris, France.No affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article

Language

eng

PubMed ID

22365767

Citation

Fischer, Alain, et al. "Strategies for Retrovirus-based Correction of Severe, Combined Immunodeficiency (SCID)." Methods in Enzymology, vol. 507, 2012, pp. 15-27.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID). Meth Enzymol. 2012;507:15-27.
Fischer, A., Hacein-Bey-Abina, S., & Cavazzana-Calvo, M. (2012). Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID). Methods in Enzymology, 507, 15-27. https://doi.org/10.1016/B978-0-12-386509-0.00002-8
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Strategies for Retrovirus-based Correction of Severe, Combined Immunodeficiency (SCID). Meth Enzymol. 2012;507:15-27. PubMed PMID: 22365767.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID). AU - Fischer,Alain, AU - Hacein-Bey-Abina,Salima, AU - Cavazzana-Calvo,Marina, PY - 2012/2/28/entrez PY - 2012/3/1/pubmed PY - 2012/6/12/medline SP - 15 EP - 27 JF - Methods in enzymology JO - Meth. Enzymol. VL - 507 N2 - Severe combined immunodeficiencies (SCIDs) appear as optimal disease targets to challenge potential efficacy of gene therapy. Ex vivo, retrovirally mediated gene transfer into hematopoietic progenitor cells has been shown to provide sustained correction of two forms of SCID, that is, SCID-X1 and adenosine deaminase deficiencies. In the former case, however, genotoxicity was observed in a minority of patients as a consequence of retroviral integration into proto-oncogenes loci and transactivation. Design of vectors in which the enhancer element of retroviral LTR has been deleted and an internal promoter added (self-inactivated vectors) could provide both safe and efficient gene transfer as being presently tested. SN - 1557-7988 UR - https://www.unboundmedicine.com/medline/citation/22365767/Strategies_for_retrovirus_based_correction_of_severe_combined_immunodeficiency__SCID__ L2 - https://linkinghub.elsevier.com/retrieve/pii/B978-0-12-386509-0.00002-8 DB - PRIME DP - Unbound Medicine ER -