In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies
Author:
Publisher
American Society for Clinical Investigation
Subject
General Medicine
Link
https://insight.jci.org/articles/view/162939/files/pdf
Reference58 articles.
1. Adenovirus vectors in hematopoietic stem cell genome editing
2. In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors
3. In vivo HSC transduction in rhesus macaques with an HDAd5/3+ vector targeting desmoglein 2 and transiently overexpressing cxcr4
4. Hematopoietic Stem Cell and Its Bone Marrow Niche
5. A Combined In Vivo HSC Transduction/Selection Approach Results in Efficient and Stable Gene Expression in Peripheral Blood Cells in Mice
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1. Drug delivery systems for CRISPR-based genome editors;Nature Reviews Drug Discovery;2023-09-18
2. Current approaches and potential challenges in the delivery of gene editing cargos into hematopoietic stem and progenitor cells;Frontiers in Genome Editing;2023-09-15
3. Drug delivery systems for CRISPR-based genome editors;NAT REV DRUG DISCOV;2023
4. Hope on the Horizon: New and Future Therapies for Sickle Cell Disease;Journal of Clinical Medicine;2023-09-01
5. The p53 challenge of hematopoietic stem cell gene editing;Molecular Therapy - Methods & Clinical Development;2023-09
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