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Project: Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System

PI:   Benjamin E Deverman, PhD NIH Report

This project aims to advance the NIH Somatic Cell Genome Editing Program’s objective to identify novel delivery technologies that enable genome editing in therapeutically relevant somatic cell populations. We will use proven virus engineering methods to develop new vehicles that can deliver genome editing machinery throughout the adult mammalian central nervous system. Accomplishing this objective would pave the road for applying gene editing, and gene therapy more broadly, to the study and treatment of neurological and psychiatric disorders.
Summary of data submissions:
  • Data for 2  experiments were submitted on 2021-04-17 SCGE ID:1036
  • 1 of 2 experiments have been validated

Submissions Details

SCGE ID:1036 - Submission Date: 2021-04-17 
Experiment Name Type Description
Selection of gRNA sequences and gRNA scaffold modification lead to improved editing of the Ai9 locus in vitro

In Vitro Reporter transgene activation by SaCas9 gRNA target and modified scaffold sequences by transient transfection in immortalized Ai9 mouse fibroblasts
Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.

In Vivo 3e11 vg/mouse of AAV-BI28:GFAP-SaCas9-WPRE-pA and 3e11 vg/mouse of AAV-BI28:GFAP-NLS-GFP-U6-L1-U6-R2 were codelivered intravenously to adult male and female Ai9 mice. Editing was assessed in brain sections 4 weeks later.