Chronic enzyme replacement therapy ameliorates neuropathology in alpha-mannosidosis mice.

ABSTRACT:

Objective

The lysosomal storage disease alpha-mannosidosis is caused by the deficiency of the lysosomal acid hydrolase alpha-mannosidase (LAMAN) leading to lysosomal accumulation of neutral mannose-linked oligosaccharides throughout the body, including the brain. Clinical findings in alpha-mannosidosis include skeletal malformations, intellectual disabilities and hearing impairment. To date, no curative treatment is available. We previously developed a beneficial enzyme replacement therapy (ERT) regimen for alpha-mannosidase knockout mice, a valid mouse model for the human disease. However, humoral immune responses against the injected recombinant human alpha-mannosidase (rhLAMAN) precluded long-term studies and chronic treatment.

Methods

Here, we describe the generation of

SUBMITTER: Damme M 

PROVIDER: S-EPMC4693626 | biostudies-literature | 2015 Nov

REPOSITORIES: biostudies-literature