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Successes and challenges in clinical gene therapy

1 Nov 2023

Abstract excerpt

Despite the ups and downs in the field over three decades, the science of gene therapy has continued to advance and provide enduring treatments for increasing number of diseases. There are active clinical trials approaching a variety of inherited and acquired disorders of different organ systems. Approaches include ex vivo modification of hematologic stem cells (HSC), T lymphocytes and other immune cells, as well...

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PMID: 37935854
DOI: 10.1038/s41434-023-00390-5

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