Summary

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Projects & Experiments

This Delivery System: AAV 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)
Vascularized kidney organoids on chip for efficacy and toxicity testing of somatic genome editing Experiments AAV tropism in kidney organoids cultured under flow Biomarker assays to evaluate toxicity and inflammation induced by AAVs in ES cell derived kidney organoids. Biomarker assays to evaluate toxicity induced by AAVs in iPS cell derived kidney organoids. AAV2/2 transduction efficiencies in kidney organoids cultured under flow determined by FACS Delivery of saCas9 and gRNAs to nephron epithelia by AAV2 in kidney organoids. Evaluation of DNA damage, cellular toxicity and inflammatory markers following saCas9 and gRNAs delivery by AAV2 in kidney organoids. AAV tropism in kidney organoids derived from human pluripotent stem cells.	Biological Effects Initiative	Ryuji Morizane, MD, PhD (Massachusetts General Hospital)
Evolving High Potency AAV Vectors for Neuromuscular Genome Editing Experiments Cre Recombinase dose escalation study in Ai9 mice	Delivery Systems Initiative	Aravind Asokan, PhD (Duke University)