T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient ... View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

1998-07

AUTHORS

D B Kohn, M S Hershfield, D Carbonaro, A Shigeoka, J Brooks, E M Smogorzewska, L W Barsky, R Chan, F Burotto, G Annett, J A Nolta, G Crooks, N Kapoor, M Elder, D Wara, T Bowen, E Madsen, F F Snyder, J Bastian, L Muul, R M Blaese, K Weinberg, R Parkman

ABSTRACT

Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for gene-corrected T lymphocytes, which may overcome inefficient gene transfer. Four years after three newborns with this disease were given infusions of transduced autologous umbilical cord blood CD34+ cells, the frequency of gene-containing T lymphocytes has risen to 1-10%, whereas the frequencies of other hematopoietic and lymphoid cells containing the gene remain at 0.01-0.1%. Cessation of polyethylene glycol-conjugated adenosine deaminase enzyme replacement in one subject led to a decline in immune function, despite the persistence of gene-containing T lymphocytes. Thus, despite the long-term engraftment of transduced stem cells and selective accumulation of gene-containing T lymphocytes, improved gene transfer and expression will be needed to attain a therapeutic effect. More... »

PAGES

775-780

Identifiers

URI

http://scigraph.springernature.com/pub.10.1038/nm0798-775

DOI

http://dx.doi.org/10.1038/nm0798-775

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1042866864

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/9662367


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