Table 1.

Genome editing in the mdx mouse. Summary of treatment and outcomes in three recent studies of in vivo genome editing with CRISPR/Cas9 delivered by AAV vectors for the correction of DMD in the mdx mouse model

Type of injection	Long et al.			Nelson et al.			Tabebordbar et al.
	IM	RO	IP	IM to TA	Tail vein	IP	IM to TA	Tail vein	IP
Administration age	P12	P18	P1	8 weeks	8 weeks	P2	6 weeks	6 weeks	P3
Cas9	Streptococcus pyogenes			Staphylococcus aureus			Staphylococcus aureus
AAV serotype	9			8			8
Dose	1E13	1.8E13	6E12	1E12	5.4E12	5.6E11	1.5E12	3.6E13	3E12
Treatment duration (weeks)	3, 6	4, 8, 12	4, 8	8, 24	8	7	4	6	3
% Modification in gDNA				2			2
% Modification in cDNA				59			39
% Dystrophin restoration by Western				8			3-8
% Dystrophin-positive fibers by IHC	53	27	23	67
Muscle function improved	Grip strength In situ TA force						In situ TA force
Cardiac cell editing		Yes			Yes				Yes
Satellite cell editing							Yes

IM intramuscular, RO retroorbital, IP intraperitoneal, P days post-natal, TA tibialis anterior muscle