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Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.
Lawal, R AdeBisi; Walters, Mark C; Fitzhugh, Courtney D.
Affiliation
  • Lawal RA; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 4-5140, Bethesda, MD 20892, USA; Hematology Branch, National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA.
  • Walters MC; University of California San Francisco Benioff Children's Hospital, 747 52nd Street, Oakland CA 94609, USA.
  • Fitzhugh CD; Cellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institute, National Institutes of Health, 10 Center Drive, Building 10, Room 6N240A, Bethesda, MD 20892, USA. Electronic address: fitzhughc@nhlbi.nih.gov.
Hematol Oncol Clin North Am ; 36(6): 1313-1335, 2022 12.
Article in En | MEDLINE | ID: mdl-36400545
ABSTRACT
Curative therapies for sickle cell disease (SCD) include allogeneic human leukocyte antigen (HLA)- matched sibling and haploidentical hematopoietic cell transplant (HCT), gene therapy, and gene editing. However, comparative trial data that might facilitate selecting one curative therapy over another are unavailable. New strategies to decrease graft rejection and graft-versus-host disease (GVHD) risks are needed to expand haploidentical HCT. Myeloablative gene therapy and gene editing also has limitations. Herein, we review recent studies on curative therapies for SCD in the past 5 years.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cell Transplantation / Graft vs Host Disease / Anemia, Sickle Cell Type of study: Etiology_studies Limits: Humans Language: En Journal: Hematol Oncol Clin North Am Journal subject: HEMATOLOGIA / NEOPLASIAS Year: 2022 Document type: Article Affiliation country:
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cell Transplantation / Graft vs Host Disease / Anemia, Sickle Cell Type of study: Etiology_studies Limits: Humans Language: En Journal: Hematol Oncol Clin North Am Journal subject: HEMATOLOGIA / NEOPLASIAS Year: 2022 Document type: Article Affiliation country: