Table 5.

Summary of gene editing to successfully improve hearing function in mouse models.^{[ 25 ]}

Gene	Mouse Model	Injection Time	Delivery Route	Vector‐Promoter	Dosage	Expression Site
	Gene knockout
Tmc1	Tmc1 Bth/+	P0‐2[ 87 ]	Cochleostomy	Lipofectamine 2000	0.3 uL, Cas9+sgRNA: 25µm	HCs
		P1[ 88 ]	Inner ear	Anc80L65‐CMV	1 µL, 4.8 × 1014vg mL−1
		P1[ 73 ]	utricle	PHP.B‐CMV	1 µL, 5.4 × 1014vg mL−1 + 5.8 × 1014vg mL−1
Myo6	Myo6 C442Y/+[ 89 ]	P0‐2	SM	PHP.eB‐EFS	0.5 µL, 3.73 × 1013vg mL−1	HCs
Kcnq4	Kcnq4 W276S/+[ 90 ]	P1‐3	Utricle, PSCC, RWM, SM	Anc80L65‐CMV	1 µL, 1 × 1012vg mL−1	OHCs
	Kcnq4 G299D /+[ 91 ]	P1‐2	SM	PHP.eB‐CMV	0.5 µL, 1.16 × 1013vg mL−1 + 2 × 1013vg mL−1
Pcdh15	Pcdh15 av‐3J[ 92 ]	P0‐2	SM	AAV2/9‐CMV	0.6 µL, 1.5 × 1014vg mL−1	HCs
	Gene correction
Klhl18	Klhl18 F lowf [ 93 ]	P1	Inner ear	PHP.eB‐EF1α	0.5 µL, 2 × 1013vg mL−1 + 1 × 1014vg mL−1	IHCs
Tmc1	Tmc1 Baringo[ 94 ]	P1	Inner ear	Anc80L65‐Cbh	1 µL, 6.11 × 1012vg mL−1 + 8.26 × 1012vg mL−1	HCs
Myo6	Myo6 C442Y/+[ 95 ]	P0‐2	SM	PHP.eB‐EFS	0.5 or 0.3584 µL, 1.43 × 1010vg	HCs
Otof	OtofQ829X/Q829X [ 8 ]	P0‐3, P5‐7, P30	SM, RWM	AAV9‐CMV	0.5 µL, 1 µL, 1–1.5 µL, 1.25 × 1010vg, 1.25 × 1010vg, 9.21 × 1010vg	IHCs

RWM: round window membrane, SM: Scala media, PSCC: posterior semicircular canal, HCs: hair cells, IHCs: inner hair cells. OHCs: outer hair cells, VG: vector genomes.