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...

पूर्ण विवरण

में बचाया:
ग्रंथसूची विवरण
मुख्य लेखकों: 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.
स्वरूप: Artigo
भाषा:Inglês
प्रकाशित: American Society of Hematology 2007
विषय:
Gene Therapy
ऑनलाइन पहुंच: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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