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Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overview

31 Dec 2024

Abstract excerpt

Gene therapy (GT) as a groundbreaking approach holds promise for treating many diseases including immune deficiencies and blood disorders. GT can benefit patients suffering from these diseases, especially those without matched donors or who are at risk after hematopoietic stem cell transplantation (HSCT). Due to all the advances in the field of GT, its main challenge is still gene delivery. Generally, gene...

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PMID: 39844821
DOI: 10.1016/j.reth.2024.12.007

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