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Updated: May 7, 2025

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
Published on: February 15, 2019
All-in-one gene therapy alternative for DBAS
1Division of Hematopoietic Innovative Therapies, CIEMAT, Madrid, Spain; Instituto Nacional de Investigación Biomédica en Enfermedades Raras (CIBERER), Madrid, Spain; Advanced Therapies Unit, IIS-Fundación Jimenez Diaz (IIS-FJD, UAM), 28040 Madrid, Spain.
Abstract:
Diamond-Blackfan anemia syndrome is a ribosomopathy classified among the bone marrow failure syndromes. This disease exhibits significant heterogeneity, with up to 24 genetic variants identified to date. Voit et al.1 present compelling preclinical evidence supporting a universal lentiviral gene therapy strategy to treat patients, regardless of the specific gene involved.
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