An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency Journal Article

  


Authors: Ferrari, G.; Rossini, S.; Giavazzi, R.; Maggioni, D.; Nobili, N.; Soldati, M.; Ungers, G.; Mavilio, F.; Gilboa, E.; Bordignon, C.
Article Title: An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency
Abstract: 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.
Keywords: controlled study; human cell; nonhuman; polymerase chain reaction; mouse; animal; mice; animal experiment; mice, mutant strains; gene transfer; animalia; genetic vectors; gene therapy; base sequence; lymphocytes; combined immunodeficiency; immunologic deficiency syndromes; phosphotransferases; retroviridae; oligonucleotides; peripheral lymphocyte; lymphocyte transfusion; adenosine deaminase; recombinant dna; human; priority journal; article; kanamycin kinase; support, non-u.s. gov't; ada
Journal Title: Science
Volume: 251
Issue: 4999
ISSN: 0036-8075
Publisher: American Association for the Advancement of Science  
Date Published: 1991-03-15
Start Page: 1363
End Page: 1366
Language: English
DOI: 10.1126/science.1848369
PUBMED: 1848369
PROVIDER: scopus
DOI/URL:

https://www.scopus.com/inward/record.uri?eid=2-s2.0-0025811758&doi=10.1126%2fscience.1848369&partnerID=40&md5=86cf849779e01de4afbad4ffe45d2080
Notes: Source: Scopus
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MSK Authors
  1. Grace U. Pedersen
    16 Pedersen
  2. Eli Gilboa
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