Table 4.

Summary of gene replacement for improving the hearing function in mouse models.^{[ 25 ]}

Gene	Mouse Model	Injection Time	Delivery Route	Vector‐Promoter	Dosage	Expression Site
Vglut3	Vglut3−/−	P0‐P3, P10‐P12[ 4 ]	RWM, cochleostomy	AAV1‐AM/CBA	0.6–1 µL, 2.3 × 1013 vg mL−1	IHCs
		5, 8 and 20 weeks[ 67 ]	PSCC	AAV8‐CMV	1 µL, 2.04 × 1013 vg mL−1
Gjb6	Gjb6−/ −[ 68 ]	E11.5	Otocysts	Plasmid	3 µg/µL,/	SL, SV, SLi, SCs
Gjb2	P0cre‐Cx26fl/fl [ 69 ]	P0, P42	RWM	AAV1‐CMV	/, 8.6 × 1011 vg	SL, SV, SLi, SCs
Kcnq1	Kcnq1−/ −[ 70 ]	P0‐2	SM, RWM	AAV1‐CBA	0.5 µL, 5.2 × 1012–1.5 × 1013 vg mL−1	SV
Pjvk	Pjvk−/ −[ 71 ]	P3	RWM	AAV8‐CAG	2 µL, 1 × 1013 vg mL−1	HCs
Tmc1	Tmc1−/− , Tmc1 Bth/+[ 48 ]	P0‐2	RWM	AAV1‐CBA	1 µL, 1.8–2.4 × 1013 vg G mL−1	HCs
	Tmc1−/ −[ 72 ]	P1‐2	RWM	Anc80L65‐CMV	1 µL, 1.4 × 1014 vg mL−1
	Tmc1−/− , Tmc1 Baringo[ 73 ]	P1, P7	Utricle	PHP.B‐CMV	1 µL, 5.46 × 1010vg,
Msrb3	Msrb3−/ −[ 74 ]	E12.5	Otocytst	AAV1‐CMV	0.6–1 µL, 1.31 × 1013 vg mL−1	HCs
Ush1c	Ush1c c.216G>A[ 75 ]	P0‐1, P10‐12	RWM	Anc80 L65‐CMV	0.8–1 µL, 1.31 × 1013 vg mL−1	HCs
Ush1g	Ush1g−/ −[ 76 ]	P2.5	RWM	AAV8‐CAG	2 µL, 1.47 × 1013 vg mL−1	HCs
Whirlin	Whrn wi/wi[ 77 ]	P4	PSCC	AAV8‐CMV	0.98 µL 1 × 1013 vg mL−1	HCs
Clrn1	Clrn1ko/ko −[ 78 ]	P1‐3	RWM	AAV2/AAV8‐smSBA	2 µL, 8.6 × 1012 vg mL−1, 3.41 × 1013 vg mL−1	HCs
	Clrn1fl/fl Myo15Cer, Clrn1ex4 − / [ 79 ]	P1‐3	RWM	AAV8‐CAG	2 µL, 6 × 1012 vg mL−1
	Clrn1−/ −[ 66 ]	P0‐P1	RWM	PHP.B‐CBA	1‐1.2 µL, 7.4 × 1010 vg
Lhfpl5	Lhfpl5−/ −[ 80 ]	P1‐2	RWM	Exosome‐AAV1‐CBA	1‐1.2 µL, 2.7 × 109 vg	HCs
Otof	Otof−/−	P6‐7[ 81 ]	RWM	AAV2/6‐CMV	1, 1.2 × 1010 vg or 1.38 × 1010 vg	IHCs
		P10, 17, 30[ 10 ]		AAV2 quadY‐F‐CMV	2 µL, 6.3 × 1012vg mL−1 + 4.5 × 1012 vg mL−1
		P0‐2, P30[ 7 ]		PHP.eB‐CAG	2 µL 2/0.5 × 1010 vg
		P0‐2, P30[ 82 ]		PHP.eB‐CAG/Myo15	2 µL, 1 × 1010 vg
		P0‐2, P30[ 6 ]		AAV1‐Myo15	/, 7.5 × 109 vg, 1.5/3/6 × 1010 vg
	OTOFQ939*/Q939*	P30[ 5 ]	PSCC	Anc80L65‐Myo15	1–2 µL, 2.8 × 1010 vg
Slc26a4	Slc26a4tm1Dontuh/tm1Dontuh, Slc26a4−/ −[ 83 ]	E12.5	Otocyst	AAV2/1‐CMV	0.6–1 µL, 1.08 × 1013 vg mL−1	Outer sulcus
Strc	Strc−/ −[ 84 ]	P0‐1	Utricle	PHP.B‐CMV	1 µL,1.57 × 1011 vg mL−1 + 3.1 × 1011 vg mL−1	OHCs
Kcne1	Kcne1−/ −[ 85 ]	P0‐2	PSCC	AAV1‐CBA	Low dose: 0.5‐1 µL, high dose: 1.5–2 µL, 1.5 × 1013 vg mL−1	SV
Syne4	Syne4−/ −[ 86 ]	P0‐1.5	PSCC	PHP.B‐CMV	1 µL, 7.7 × 1012 vg mL−1	OHCs
Pcdh15	Myo15cre‐Pcdh15fl/fl[ 13 ]	P0‐1	RWM	PHP.B‐CMV	/, 5 × 1010 vg	HCs

SM: Scala media, RWM: round window membrane, PSCC: posterior semicircular canal, HC: hair cell, SL: spiral limbus, SV: stria vascularis, SLi: spiral ligament, IHC: inner hair cell. OHC: outer hair cell, VG: vector genomes.