Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

A patient with adenosine deaminase–deficient severe combined immune deficiency (ADA-SCID) was enrolled in a study of retroviral-mediated ADA gene transfer to bone marrow hematopoietic stem cells. After the discontinuation of ADA enzyme replacement, busulfan (75 mg/m(2)) was administered for bone mar...

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Autori principali: Engel, Barbara C., Podsakoff, Greg M., Ireland, Joanna L., Smogorzewska, E. Monika, Carbonaro, Denise A., Wilson, Kathy, Shah, Ami, Kapoor, Neena, Sweeney, Mirna, Borchert, Mark, Crooks, Gay M., Weinberg, Kenneth I., Parkman, Robertson, Rosenblatt, Howard M., Wu, Shi-Qi, Hershfield, Michael S., Candotti, Fabio, Kohn, Donald B.
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Hematology 2007
Soggetti:
Gene Therapy
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC1785101/
https://ncbi.nlm.nih.gov/pubmed/16973956
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2006-06-031476
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