Summary

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Associated Publications

Publication Title
Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. NCBI
Focused ultrasound-mediated brain genome editing. NCBI

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Projects & Experiments

This Model System: Ai9 mouse is being used ...

Project	Initiative	Contact PI
SCGE AAV Tropism Supplement: Evaluation Across Multiple Tissues in Mice Experiments AAV Tropism project	AAV tropism	William Raymond Lagor, PhD Jason D Heaney, PhD Mary E Dickinson, PhD Guang-Ping Gao, PhD Stephen A Murray, PhD Cathleen M Lutz, PhD (Baylor College of Medicine)
BCM-Rice Resource for the Analysis of Somatic Gene Editing in Mice Experiments Independent validation of Deverman delivery platform using engineered AAVs to deliver CRSIPR/Cas9 to mouse brain	Animal Reporter and Testing Center	Jason D Heaney, PhD (Baylor College of Medicine)
Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Experiments Cre Recombinase dose escalation study in Ai9 mice Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CB promoter) Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 Cas9 to sgRNA ratio (CMV promoter) and self complementary sgRNA vector. Comparing CRISPR/Cas9 gene editing efficencies between AAV9 and AAVcc47 in Ai9 mice with a 1:1 cas9 to sgRNA ratio (CMV promoter) Comparing CRISPR/Cas9 gene editing efficiencies between AAV9 and AAVcc47 in Ai9 mice with a 1:3 Cas9 to sgRNA ratio (CMV promoter)	Delivery Systems	Aravind Asokan, PhD (Duke University)
Develop Combinatorial Non-Viral and Viral CRISPR Delivery for Lung Diseases Experiments Testing AAV5 for activation of tdTomato in mouse airway club and ciliated cells Testing AAV5 for activation of tdTomato in mouse airway	Delivery Systems	Guang-Ping Gao, PhD (University of Massachusetts Medical School, Worcester)
Focused Ultrasound-mediated Delivery of Gene-editing Elements to the Brain for Neurodegenerative Disorders Experiments FUS (focused ultrasound) array validation in Ai9 mice	Delivery Systems	Kam W Leong, PhD (Columbia University Health Sciences)
Novel AAVs Engineered for Efficient and Noninvasive Cross-Species Gene Editing Throughout the Central Nervous System Experiments Testing gRNA sequence and gRNA scaffold modified in Ai9 mice.	Delivery Systems	Benjamin E Deverman, PhD (Broad Institute)