Transcutaneous ultrasound mediated gene delivery into canine livers achieves therapeutic levels of FVIII expression.
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DOI: 10.1182/bloodadvances.2021006016
Abstract: A safe, effective, and inclusive gene therapy will significantly benefit a large population of hemophilia patients. We employed a minimally invasive transcutaneous ultrasound mediated gene delivery (UMGD) strategy combined with microbubbles (MBs) to enhance gene… read more here.
Keywords: transcutaneous ultrasound; canine livers; mediated gene; gene ... See more keywords