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Project: Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders

PI:   Kam W Leong, PhD  NIH Report

Gene editing may offer a new therapeutic strategy to tackle many neurodegenerative disorders that remain untreatable. Current methodologies of delivering CRISPR-based gene editing elements to the brain are highly inefficient. We propose to develop a noninvasive, efficient approach to achieve gene editing in the brain using focused ultrasound technology.
Summary of data submissions:
  • Data for 1  experiments were submitted on 2022-04-15 SCGE ID:1060

Submissions Details

SCGE ID:1060 - Submission Date: 2022-04-15 
Experiment Name Type Description
FUS (focused ultrasound) array validation in Ai9 mice

 In Vivo 9.3 week-old Ai9 mice (4 male and 4 female) were administered Ai9-targeting SaCas9 AAV9 vector through intravenous adminsitration (2E12 vg/mouse) and left hemisphere was targeted by FUS (focused ultrasound) array for BBB (blood brain barrier) opening