[CIS PIDD] [cis-pidd] ADA-deficient SCID

[CIS-PIDD]

I have a new ADA deficient SCID.  Three weeks old.  Perfectly healthy. No sibs.
We have one 6/6 cord blood donor, and ten 10/10 bone marrow donor options (<40 years old) from the NMDP.

Would people transplant now without a prep regimen, transplant now with RIC, give PEG-ADA for 2 years and then transplant when conditioning toxicity might be less, or try gene therapy?  (If the latter, who has an active program?


Howard


Howard M. Lederman, M.D., Ph.D.
Professor of Pediatrics, Medicine and Pathology
Division of Pediatric Allergy and Immunology
Johns Hopkins Hospital - CMSC 1102
600 N. Wolfe Street
Baltimore, MD 21287-3923
Phone: 410-955-5883
Fax: 410-955-0229
Email: Hlederm1 at jhmi.edu<https://mobile.johnshopkins.edu/OWA/redir.aspx?C=02aee18ab96e42ab8cb61c09ecb79487&URL=mailto%3aHlederm1%40jhem.jhmi.edu>

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