NewsMOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPYSaturday, March 7, 2026 · March 7, 2026
Comparative evaluation of liver-directed knockin strategies with viral and nonviral vectors in mouse inherited disease models.
WHY IT MATTERS
Recent peer-reviewed research on Hemophilia B that may be relevant for patients and caregivers.
CRISPR-Cas9-mediated gene knockin has emerged as a promising strategy for early-onset genetic disease intervention. However, the therapeutic efficacy and editing outcomes of different knockin strategies remain incompletely understood. Here, we systematically evaluated three major liver-directed knoc...
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