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Key Points • Correction of the sickle-cell mutation and disease phenotypes is achieved by in vivo HSC transduction with vectorized prime editors.• Our approach for in vivo HSC prime editing… Click to show full abstract
Key Points • Correction of the sickle-cell mutation and disease phenotypes is achieved by in vivo HSC transduction with vectorized prime editors.• Our approach for in vivo HSC prime editing that does not require HSC transplantation and myeloablation should simplify HSC gene therapy.
Keywords:
disease;
prime editing;
vivo hsc;
hsc prime;
sickle cell
Journal Title: Blood
Year Published: 2023
Link to full text (if available)
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Journal Title: Blood
Year Published: 2023
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!