Gene Therapy for Human Severe Combined Immunodeficiencies
نویسندگان
چکیده
منابع مشابه
Gene Therapy for Primary Immunodeficiencies
Primary immunodeficiencies (PID) are caused by mutations in genes involved in the normal development or activity of the immune system [1, 2]. PIDs include Band T-cell defects, phagocytic disorders, and complement deficiencies with the common feature of frequent lifethreatening infections. The phenotypes vary from asymptomatic (IgA deficiency) to severe PIDs (such as Severe combined immunodefici...
متن کاملEarly Diagnosis of Severe Combined Immunodeficiencies
aDirector of Research, Israelita Albert Einstein Hospital, São Paulo, SP, Brasil. Received on January 11, 2017. © 2017 Sociedade de Pediatria de São Paulo. Published by Zeppelini Publishers. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/). EARLY DIAGNOSIS OF SEVERE COMBINED IMMUNODEFICIENCIES Diagnóstico precoce das imunodeficiências combinad...
متن کاملAdvances of gene therapy for primary immunodeficiencies
In the recent past, the gene therapy field has witnessed a remarkable series of successes, many of which have involved primary immunodeficiency diseases, such as X-linked severe combined immunodeficiency, adenosine deaminase deficiency, chronic granulomatous disease, and Wiskott-Aldrich syndrome. While such progress has widened the choice of therapeutic options in some specific cases of primary...
متن کاملGene therapy of primary T cell immunodeficiencies.
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 re...
متن کاملGene therapy for severe combined immunodeficiency: are we there yet?
Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related...
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ژورنال
عنوان ژورنال: Immunity
سال: 2001
ISSN: 1074-7613
DOI: 10.1016/s1074-7613(01)00175-3