. Author manuscript; available in PMC: 2016 Jan 31.

Published in final edited form as: Mol Genet Metab. 2014 Oct 7;114(2):83–93. doi: 10.1016/j.ymgme.2014.09.011

Table II.

Correction of behavioral, hearing and vision abnormalities in MPS I mice

RV-hIDUA-treated mice [75, 113]
Test/parameter	Improvement^a		Function

	IDUA activity in Forebrain (% WT)
	23%	73%
Sensorimotor tests
1) Holding onto inverted screen	–	+	Balance, strength, motor coordination; vestibular function
2) Pole gymnastics	–	+	Balance, strength, motor coordination; vestibular function

Open-field tests
1) Walking in first 5 min	+	+	Spontaneous locomotion
2) Whole-body movements	±	+	Environmental habituation
3) Rearings (vertical activity)	–	–

Auditory test
Auditory-evoked brain-stem responses	–	±	Hearing
Vision test
Electroretinogram	–	+	Rod function

LV-transduced HSC-treated mice [115]
Test/parameter	IDUA in whole brain 2-5X WT	Function
Open-field tests
1) Rearings (vertical activity)	+	Spontaneous locomotion
2) Horizontal transitions	+	Environmental habituation
Auditory test
Auditory-evoked brain-stem responses	±	Hearing

improvement: +, complete or near-complete correction; –, no improvement; ±, partial but significant improvement