Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease.
Journal Information
Full Title: Mol Ther Methods Clin Dev
Abbreviation: Mol Ther Methods Clin Dev
Country: Unknown
Publisher: Unknown
Language: N/A
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Subject Category: Medicine, Research & Experimental
Available in Europe PMC: Yes
Available in PMC: Yes
PDF Available: No
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"Declaration of interests M.H.P. holds equity in CRISPR Tx, holds equity and serves on the SAB of Allogene Tx and Ziopharm Therapeutics, on the board of directors of Graphite Bio, and as an advisor to Versant Ventures."
"This work was supported by CIRM CLIN1-10084, 10.13039/100000002NIH grant R01HL135607, the 10.13039/100013856Debbie's Dream Foundation, the Laurie Kraus Lacob Faculty Scholar Fund in Pediatric Translational Medicine, and the Sutardja Chuk Professorship."
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Last Updated: Aug 05, 2025