DMD low gRNA-2 | SCGE Toolkit

SCGE ID	10000000341
Lab ID	DMD low gRNA-2
Source	VectorBuilder
Target Locus	DMD
Species	human
Description	One of two low-off target gRNAs delivred by dual gRNA AAV vector AAV-gRNA
Guide Compatibility	SauCas9

Target Sequence	TGTTTCCTTCCTTGTTCCCAG
Target Sequence + PAM	TGTTTCCTTCCTTGTTCCCAGCAGAAT
Position	hg38/X:31508566-31508586

Spacer Sequence	UGUUUCCUUCCUUGUUCCCAG
Spacer Length	21
Modifications	none

This Guide: DMD low gRNA-2 is being used ...

Project	Initiative	Contact PI
Vascularized kidney organoids on chip for efficacy and toxicity testing of somatic genome editing Experiments Evaluation of DNA damage, cellular toxicity and inflammatory markers following saCas9 and gRNAs delivery by AAV2 in kidney organoids. Delivery of saCas9 and gRNAs to nephron epithelia by AAV2 in kidney organoids.	Biological Effects Initiative	Ryuji Morizane, MD, PhD (Massachusetts General Hospital)

Guide: DMD low gRNA-2