Table 1.

Summary of the MPS I Animals, AAV Vector Injections, and Clinical Outcome

	Animal ID
	2		3		4		1
Animal research ID	I-709		I-710		I-712		I-744
Sex	F		M		M		F
Eye	R	L	R	L	R	L	R	L
Injection
Vector	AAV-GFP	AAV-IDUA	AAV-IDUA	AAV-GFP	AAV-GFP	AAV-IDUA	AAV-IDUA	AAV-GFP
Volume injected intrastromally (μL)	80	80	50	50	65	65	65	0
Viral dose (vg/cornea)	8 × 1010	8 × 1010	5 × 1010	5 × 1010	6.5 × 1010	6.5 × 1010	6.5 × 1010	5 × 1010
Intrastromal injection procedure	uneventful	uneventful	uneventful	uneventful	uneventful	additional intrastromal needle insertion	AC needle entry; additional intrastromal needle insertion	uneventful
Intracorneal hemorrhagea	+	+++	++	−	+	+++	−	−
Pre-injection
Age (months)	13		13		13		9
Corneal disease stage	advanced		advanced		advanced		early
Clinical scoreb	4	4	4	4	4	4	1	1
CCT (μm)	782	828	725	720	790	795	479	522
Endpoint (25 weeks post-injection)
Age (months)	19		19		19		15
Clinical scoreb	2c	1	0	4	4	0	1	3
CCT (μm)	570	629	493	867	802	543	465	583
Capsid antibody titer	1:64		−		−		−
Corneal Edema
Onset (weeks post-injection)	10	6	−	19	−	−	17	−
Duration (weeks)	3	3	−	3	−	−	2	−
Steroid treatment	topical	topical + systemic	−	topical	−	−	topical	−

AAV-GFP, AAV8G9-CMV-GFP; AAV-IDUA, AAV8G9-CMV-IDUA; AC, anterior chamber.

^aIntracorneal hemorrhage score: −, no hemorrhage; +, a small single focus of hemorrhage; ++, small multifocal hemorrhages; +++, large area of coalesced multifocal hemorrhages.

^bCornea clinical score: 0 = clear, storage (−); 1 = storage (+), minimal vascularization; 2 = storage (+), midrange vascularization; 3 = storage (+), diffuse vascularization; 4 = storage (+), diffuse vascularization, crystalline deposits, fibrosis.

^cReduced perfusion of diffuse vasculature.