Non-Viral RNA-Lipid Nanoparticles for High Efficiency Genome Editing of CD34+ Hematopoietic Stem and Progenitor Cells for Advanced Cell and Gene Therapies

Background and Aims: The application of CRISPR-Cas9 gene editing in hematopoietic stem (HSC) and progenitor cells (HSPCs) has the potential to revolutionize the treatment of genetic blood disorders by correcting disease-causing mutations. Previously, we demonstrated the utility of a novel lipid nano...

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Veröffentlicht in:	Blood 2023-11, Vol.142 (Supplement 1), p.4041-4041
Hauptverfasser:	Geczy, Reka, Park, Stella, Zhao, Rita, Swaminathan, Meenakshi, Jain, Nikita, Thomas, Anitha, Zhang, Angela, Clarke, Samuel
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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