Recent advances in systemic treatments for mucopolysaccharidosis have led t
o therapies that improve the multiple somatic features of this disease, but
the therapeutic effect on ocular manifestations such as corneal clouding i
s not satisfactory. Here, we administered an adenovirus expressing human be
ta -glucuronidase (AxCAhGUS) into the anterior chamber or intrastromal regi
on of the cornea in mice with mucopolysaccharidosis type VII (B6/MPSVII), a
nd successfully treated corneal clouding of MPSVII. When we injected AxCAhG
US into the anterior chamber of the eyes, cells expressing beta -glucuronid
ase (GUSB) were located mainly in the trabecular meshwork as well as in all
corneal regions, and subsequent pathological corrections in the cornea wer
e achieved. Widespread transgene expression was also observed when we admin
istered AxCAhGUS inside the cornea after lamellar keratotomy, and rapid eli
mination of the lysosomal storage in the corneal keratocytes occurred. Furt
hermore, intrastromal vector administration did not generate significant le
vels of anti-adenovirus neutralizing antibodies, and secondary vector admin
istration was effective. Based on these observations, we conclude that it i
s worth developing a treatment strategy for corneal clouding in mucopolysac
charidosis based on direct intraocular administration of adenoviral vectors
.