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Gene Therapy
Phase I/II
CRISPR-Cas9 Base Editing in Sickle Cell Disease
Perspectivum
629 ,
401-410 (2024)
·
10.1038/s41586-024-07122-z
·
NCT05572814
·
3:50
Mechanistic animation of adenine base editing at the HBB locus, depicting guide RNA binding, adenosine deaminase activity, and haemoglobin switching in erythroid progenitors.
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