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Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

โœ Scribed by Carbonaro, D. A.; Jin, X.; Wang, X.; Yu, X.-J.; Rozengurt, N.; Kaufman, M. L.; Wang, X.; Gjertson, D.; Zhou, Y.; Blackburn, M. R.; Kohn, D. B.


Book ID
118173729
Publisher
American Society of Hematology
Year
2012
Tongue
English
Weight
892 KB
Volume
120
Category
Article
ISSN
0006-4971

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PEG-ADA: An alternative to haploidentica
โœ Michael S. Hershfield ๐Ÿ“‚ Article ๐Ÿ“… 1995 ๐Ÿ› John Wiley and Sons ๐ŸŒ English โš– 596 KB

PEG-ADA is a long-circulating form of adenosine deaminase (ADA) that has been in use for > 8 years as replacement therapy for severe combined immunodeficiency disease due to ADA deficiency. Treatment with PEG-ADA almost completely corrects metabolic abnormalities, allowing the recovery of a variable