Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials

Details

Serval ID
serval:BIB_39E9AB4FA040
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Title
Gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency: improved retroviral vectors for clinical trials
Journal
Acta Haematol
Author(s)
Onodera M., Nelson D. M., Sakiyama Y., Candotti F., Blaese R. M.
ISSN
0001-5792 (Print)
ISSN-L
0001-5792
Publication state
Published
Issued date
1999
Volume
101
Number
2
Pages
89-96
Language
english
Notes
Onodera, M
Nelson, D M
Sakiyama, Y
Candotti, F
Blaese, R M
eng
Review
Switzerland
Acta Haematol. 1999;101(2):89-96.
Abstract
Severe combined immunodeficiency (SCID) caused by adenosine deaminase deficiency (ADA-) is the first genetic disorder to be treated with gene therapy. Since 1990 when the first trial started for 2 patients with ADA- SCID, five clinical trials enrolling 11 patients have been conducted with different clinical approaches and the results obtained from these trials have recently been reported. According to these reports, T cell-directed gene transfer was useful in the treatment of ADA- SCID whereas the retroviral-mediated gene transfer to hematopoietic stem cells was insufficient for achievement of clinical benefits. This chapter reviews several crucial problems inherent in the current retroviral technology based on the clinical data observed in these pioneering ADA gene therapy trials and presents our new retroviral vector system for the next stem cell gene therapy.
Keywords
Adenosine Deaminase/*deficiency/genetics, Animals, Clinical Trials as Topic, *Genetic Therapy, *Genetic Vectors, Humans, Retroviridae/*genetics, Severe Combined Immunodeficiency/enzymology/*therapy
Pubmed
Create date
01/11/2017 10:29
Last modification date
20/08/2019 13:29
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