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Modeling Adeno-Associated Viral Vector 6-mediated <i>In Vivo</i> Gene Delivery to Expanded Non-Mobilized Haemopoietic Stem Cells from Transfusion-dependent Thalassemia Patients in a Humanized Mouse

2025-06-18

Abstract excerpt

<h4>ABSTRACT</h4> Hematopoietic stem cells (HSC) are important targets for gene modification therapies (GMT) as they originate several serious genetic conditions including the β-haemoglobinopathies. Potentially curative ex vivo GMT pose the barriers of accessibility, myeloablation-associated morbidity and prohibitive cost. In vivo GMT using non-integrating single-strand adeno-associated viral vectors (ssAAV) ar...

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Literature Corpus work
31e824e9-7da1-5869-872e-cda888274c6a
DOI
10.1101/2025.06.17.659025
Open publication

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Modeling Adeno-Associated Viral Vector 6-mediated <i>In Vivo</i> Gene Delivery to Expanded Non-Mobilized Haemopoietic Stem Cells from Transfusion-dependent Thalassemia Patients in a Humanized MouseDOI 10.1101/2025.06.17.659025
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