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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Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2007
Témata:
Gene Therapy
On-line přístup: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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