Table 1.

Treatment groups and current clinical status of AAV-treated GM1 cats.

DurationA	Serotype	DoseB	Cat	Gender	Tx age (mos.)	Endpoint or current age (mos.)	Clinical description	± Seizures*
Long-term	AAV1	High	9-1356	F	1.9	52.8	Mild hind limb weakness, carpal hyperextension	+
			8-1483	F	1.9	41.4	Normal	+
			8-1485	F	2.0	41.2	Normal	-
		Full	9-1545	F	2.3	25.3	Deceased	-
			8-1551	F	2.1	34.6	Normal	+
	AAVrh8	Full	8-1364	M	1.6	50.5	Mild hind limb weakness	-
			8-1378	M	1.3	17.5	Deceased	-
			8-1397	F	1.7	47.9	Deceased#	+
			9-1424	F	2.9	44.9	Normal	-
			8-1435	M	2.8	44.8	Mild hind limb weakness	+
			9-1515	M	1.9	29.0	Deceased#	-
			8-1626	M	3.0	29.4	Weakness of carpi and limbs (hind/fore), pelvic ataxia, tremors	+
		One-tenth	8-1574	M	1.8	21.2	Deceased	+
			8-1578	F	1.8	13.9	Deceased	-
			8-1576	F	1.8	14.2	Deceased	-
			9-1620	F	2.3	19.1	Deceased	-
Short-term (16 weeks)	AAV1	Full	9-1553	F	2.3	N/A	Normal	-
			9-1555	F	2.3	N/A	Normal	-
			8-1617	M	2.5	N/A	Normal	-
	AAVrh8	Full	9-1494	M	1.9	N/A	Normal	-
			9-1502	M	1.8	N/A	Normal	-
			8-1525	F	1.9	N/A	Normal	-
			8-1526	F	1.9	N/A	Normal	-

^ACats in the long-term cohort were euthanized at clinical humane endpoint. Short-term cats were euthanized 16 weeks post-injection. Cats received bilateral injections of the thalamus and DCN.

^BDoses: High, 1.2 × 10¹³ vg; Full, 3–4 × 10¹² vg; one-tenth dose, 3 × 10¹¹ vg.

^*Animal developed seizures that were well controlled by medication. The mean age of seizure onset was 20.1 ± 7.4 months. #Cat 8-1397 had normal gait at 47.9 months of age but did not recover from an anesthetic procedure and was euthanized. Cat 9-1515 was clinically normal at 29.0 months of age but did not recover properly from anesthesia for MRI and was euthanized.

Abbreviations: Tx = treatment; mos. = months; N/A = not applicable.