Tags

Type your tag names separated by a space and hit enter

Gene therapy of primary T cell immunodeficiencies.
Gene. 2013 Aug 10; 525(2):170-3.GENE

Abstract

Gene therapy of severe combined immunodeficiencies has been proven to be effective to provide sustained correction of the T cell immunodeficiencies. This has been achieved for 2 forms of SCID, i.e SCID-X1 (γc deficiency) and adenosine deaminase deficiency. Occurrence of gene toxicity generated by integration of first generation retroviral vectors, as observed in the SCID-X1 trials has led to replace these vectors by self inactivated (SIN) retro(or lenti) viruses that may provide equivalent efficacy with a better safety profile. Results of ongoing clinical studies in SCID as well as in other primary immunodeficiencies, such as the Wiskott Aldrich syndrome, will be thus very informative.

Authors+Show Affiliations

INSERM U768, Paris, France. alain.fischer@inserm.frNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Review

Language

eng

PubMed ID

23583799

Citation

Fischer, Alain, et al. "Gene Therapy of Primary T Cell Immunodeficiencies." Gene, vol. 525, no. 2, 2013, pp. 170-3.
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Gene therapy of primary T cell immunodeficiencies. Gene. 2013;525(2):170-3.
Fischer, A., Hacein-Bey-Abina, S., & Cavazzana-Calvo, M. (2013). Gene therapy of primary T cell immunodeficiencies. Gene, 525(2), 170-3. https://doi.org/10.1016/j.gene.2013.03.092
Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M. Gene Therapy of Primary T Cell Immunodeficiencies. Gene. 2013 Aug 10;525(2):170-3. PubMed PMID: 23583799.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Gene therapy of primary T cell immunodeficiencies. AU - Fischer,Alain, AU - Hacein-Bey-Abina,Salima, AU - Cavazzana-Calvo,Marina, Y1 - 2013/04/10/ PY - 2013/01/11/received PY - 2013/03/13/revised PY - 2013/03/14/accepted PY - 2013/4/16/entrez PY - 2013/4/16/pubmed PY - 2013/9/21/medline KW - ADA KW - GVHD KW - Gene therapy KW - HSC KW - HSCT KW - IPS KW - LV KW - Lentivirus KW - PIDs KW - RV KW - Retrovirus KW - SAE KW - SCID KW - SIN KW - Severe combined immune deficiencies KW - WAS KW - Wiskott Aldrich syndrome KW - Wiskott–Aldrich syndrome KW - adenosine deaminase KW - graft versus host disease KW - hematopoietic stem cell KW - hematopoietic stem cell transplantation KW - induced pluripotent cells KW - primary immunodeficiencies KW - retrovirus KW - self inactivated KW - serious adverse event KW - severe combined immunodeficiency SP - 170 EP - 3 JF - Gene JO - Gene VL - 525 IS - 2 N2 - Gene therapy of severe combined immunodeficiencies has been proven to be effective to provide sustained correction of the T cell immunodeficiencies. This has been achieved for 2 forms of SCID, i.e SCID-X1 (γc deficiency) and adenosine deaminase deficiency. Occurrence of gene toxicity generated by integration of first generation retroviral vectors, as observed in the SCID-X1 trials has led to replace these vectors by self inactivated (SIN) retro(or lenti) viruses that may provide equivalent efficacy with a better safety profile. Results of ongoing clinical studies in SCID as well as in other primary immunodeficiencies, such as the Wiskott Aldrich syndrome, will be thus very informative. SN - 1879-0038 UR - https://www.unboundmedicine.com/medline/citation/23583799/Gene_therapy_of_primary_T_cell_immunodeficiencies_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0378-1119(13)00361-2 DB - PRIME DP - Unbound Medicine ER -