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发表于 2025-3-30 11:26:35
Haploidentical Hematopoietic Cell Transplantation for Sickle Cell Disease of patients have such a donor. Haploidentical (i.e., half human leukocyte antigen (HLA)-matched relatives) could potentially solve this donor issue and thus provide a treatment option for essentially every patient. Ongoing investigation using either in vivo or ex vivo T-cell depletion strategies ha
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发表于 2025-3-30 16:26:07
Gene Therapy: The Path Toward Becoming a Realistic Cure for Sickle Cell Disease the only currently available curative option for patients with SCD. Data suggests an overall survival of >95% with a myeloablative regimen using HLA-matched sibling donors and >90% disease-free survival using a non-myeloablative regimen, yet less than 15% of patients with SCD have an appropriately
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发表于 2025-3-30 20:13:51
Book 2018 reviews new data about risk prediction for severe SCD, outlines the unique challenges of HSCT for patients with SCD, profiles the supportive care guidelines for patients who are undergoing HSCT, highlights our current understanding of the best transfusion support for SCD patients prior to, during