Table 3. Design of the 6-month safety study in Rs1-KO mice and the number of animals tested at each time point for any vector dose and vehicle.

Examinations	BaselineDose (vg/eye)			3rd dayDose (vg/eye)			1st monthDose (vg/eye)			6th monthDose (vg/eye)
	2E9	2E10	Veh	2E9	2E10	Veh	2E9	2E10	Veh	2E9	2E10	Veh
In-life
Ophthalmic examination	54	54	54	54	54	54	37	37	37	21	21	21
Clinical pathology	N	N	N	4	4	4	4	4	4	4	4	4
Anti-AAV8 capsid Abb	N	N	N	5	5	5	N	N	N	9	9	8
Anti-hRS Ab, hRS protein	N	N	N	5	5	5	N	N	N	9	9	8
PBMC	N	N	N	N	N	N	4	4	4	4	4	4
Clinical observationa	54	54	54	54	54	54	37	37	37	21	21	21
Body weighta	54	54	54	54	54	54	37	37	37	21	21	21
Food consumptiona	54	54	54	54	54	54	37	37	37	21	21	21
Postmortem
Vector biodistribution	N	N	N	5	5	5	N	N	N	5	5	5
hRS gene expression	N	N	N	5	5	5	N	N	N	5	5	5
Organ pathology	N	N	N	12	12	12	12	12	12	12	12	12
Ocular pathology	N	N	N	12	12	12	12	12	12	12	12	12

N, none; PBMC, peripheral blood mononuclear cell; Veh, vehicle.

^aPerformed weekly for the entire 9-month period.

^bNot all plasma samples were analyzed for AAV8 neutralizing antibody due to hemolysis in some samples.