Gene Therapy with Cord Blood Hematopoietic Stem Cells for Adenosine Deaminase Deficiency: An Update View Full Text


Ontology type: schema:Chapter     


Chapter Info

DATE

2000

AUTHORS

D. B. Kohn , K. Weinberg , J. Nolta , G. Crooks , R. Parkman

ABSTRACT

Gene therapy for ADA deficiency has been a model disease in which to evaluate hematopoietic stem cells for clinical gene therapy [1]. ADA deficiency has been a candidate disease becauseADA is a single polypeptide protein,a clinically normal phenotype is associated with a wide range of enzymatic function (5–500% of normal), andADA is widely expressed so that tissue specific expression is not required. ADA is a single polypeptide protein, a clinically normal phenotype is associated with a wide range of enzymatic function (5–500% of normal), and ADA is widely expressed so that tissue specific expression is not required. More... »

PAGES

287-291

Book

TITLE

Transplantation in Hematology and Oncology

ISBN

978-3-642-64041-4
978-3-642-59592-9

Author Affiliations

Identifiers

URI

http://scigraph.springernature.com/pub.10.1007/978-3-642-59592-9_35

DOI

http://dx.doi.org/10.1007/978-3-642-59592-9_35

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1041346381


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