Publication Date:
1991-03-15
Description:
Deficiency of adenosine deaminase (ADA) results in severe combined immunodeficiency (SCID), a candidate genetic disorder for somatic cell gene therapy. Peripheral blood lymphocytes from patients affected by ADA- SCID were transduced with a retroviral vector for human ADA and injected into immunodeficient mice. Long-term survival of vector-transduced human cells was demonstrated in recipient animals. Expression of vector-derived ADA restored immune functions, as indicated by the presence in reconstituted animals of human immunoglobulin and antigen-specific T cells. Retroviral vector gene transfer, therefore, is necessary and sufficient for development of specific immune functions in vivo and has therapeutic potential to correct this lethal immunodeficiency.〈br /〉〈span class="detail_caption"〉Notes: 〈/span〉Ferrari, G -- Rossini, S -- Giavazzi, R -- Maggioni, D -- Nobili, N -- Soldati, M -- Ungers, G -- Mavilio, F -- Gilboa, E -- Bordignon, C -- New York, N.Y. -- Science. 1991 Mar 15;251(4999):1363-6.〈br /〉〈span class="detail_caption"〉Author address: 〈/span〉Laboratory of Hematology, Istituto Scientifico H.S. Raffaele, Milano, Italy.〈br /〉〈span class="detail_caption"〉Record origin:〈/span〉 〈a href="http://www.ncbi.nlm.nih.gov/pubmed/1848369" target="_blank"〉PubMed〈/a〉
Keywords:
Adenosine Deaminase/*deficiency/genetics
;
Animals
;
Base Sequence
;
Genetic Therapy
;
Genetic Vectors
;
Humans
;
Immunologic Deficiency Syndromes/*genetics/therapy
;
Kanamycin Kinase
;
Lymphocyte Transfusion
;
Lymphocytes/physiology
;
Mice
;
Mice, Mutant Strains
;
Oligonucleotides/chemistry
;
Phosphotransferases/genetics
;
Polymerase Chain Reaction
;
Retroviridae/genetics
Print ISSN:
0036-8075
Electronic ISSN:
1095-9203
Topics:
Biology
,
Chemistry and Pharmacology
,
Computer Science
,
Medicine
,
Natural Sciences in General
,
Physics
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