Transforming clinical apheresis waste into a renewable source of patient-derived CD34+ hematopoietic stem cell biobank for beta-hemoglobinopathy research and therapeutic discovery
Clinical apheresis waste is used to generate a single-center biobank of high-quality, research-grade CD34+ HSPCs from patients with sickle cell disease and beta-thalassemia, addressing a recognized gap in the hemoglobinopathy pipeline.
Data support initiation of a first-in-human phase I trial of CRISPR_SCD001 for sickle patients presenting with severe pathology, and generates a clinical-grade, cryopreserved CD34+ cell product that corrects the sickle mutation, restores anti-sickling hemoglobins, and meets pre-clinical safety criteria.
M. DeWitt, Beatriz Campo-Fernández, Sohini Roy et al.· Molecular Therapy· 0 citations
Protocols to generate developmentally primitive and definitive RBCs from iPSCs are outlined, and methods to perform antibody identification using iPSC-derived RBCs as reagent cells in gel agglutination assays commonly used by blood banks and reference immunohematology laboratories are outlined.
Hyun Hyung An, Giulia Pavani, Stella T. Chou· Methods in molecular biology· 0 citations
PURPOSE OF REVIEW
To review advances from the past 18 months in allogeneic hematopoietic stem cell transplantation (HSCT) and autologous gene-modified stem cell transplantation for sickle cell disease (SCD), and to relate these developments to stem cell biology, conditioning and emerging prenatal strategies.
RECENT F...
Giula Mackina, Panicos Shangaris· Current opinion in hematolog...· 0 citations
Recent developments and refinements in gene transfer and editing technologies for HSPCs are reviewed, while also discussing the critical limitations and hurdles to clinical translation, as recently presented at the New Investigator Committee Gene Therapy webinar.
Kohei Shiroshita, A. Stolz, C. Malouf et al.· Experimental Hematology· 0 citations
A novel strategy for generating bioengineered HLA class I-negative, "antigen-specific" megakaryocytes (MKs) from induced pluripotent stem cells (iPSCs) is described, which allows for rapid detection and characterization of platelet-specific antibodies present in patient plasma.
Nan-Yan Zhang, Peter J Newman· Methods in molecular biology· 0 citations
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