Two is better than one: Advancing gene therapy protocols for enhanced safety and efficacy.
editorial · Level V
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- Record sourced from PubMed, PMID 39919720.
- Also identified by DOI 10.1016/j.stem.2024.12.009.
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Abstract
In this issue of Cell Stem Cell, two complementary studies from Zeng et al.<sup>1</sup> and Demirci et al.<sup>2</sup> improve the efficacy of gene therapy for β-hemoglobinopathies using multiplex CRISPR-Cas9 editing. The authors also optimize editing and conditioning protocols to reduce risks associated with current gene therapy procedures.
Medical subject headings
- Genetic Therapy