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Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome
- Source :
- Blood; 20230101, Issue: Preprints
- Publication Year :
- 2023
-
Abstract
- •Reconstitution of platelets and myeloid function correlated strongly with the number of vector copies in individual transduced colony-forming units.•Patients with poor reconstitution of Treg and Breg compartments may be at risk of ongoing autoimmunity despite high-level gene marking.
Details
- Language :
- English
- ISSN :
- 00064971 and 15280020
- Issue :
- Preprints
- Database :
- Supplemental Index
- Journal :
- Blood
- Publication Type :
- Periodical
- Accession number :
- ejs63617608
- Full Text :
- https://doi.org/10.1182/blood.2022019117