Log in with your email address username.

×

[Comment] Therapies of lysosomal storage disorders targeting the brain

Therapies for lysosomal storage disorders such as enzyme replacement have become increasingly available over the past decade. Although clinical benefit has been reported for some disease aspects, important limitations come into play when the brain is involved.1 Intrathecal rather than intravenous enzyme delivery is the focus of studies for diseases with predominant cerebral involvement, such as metachromatic leukodystrophy, but results are pending. Direct delivery of the deficient enzyme by means of stereotactic injections of recombinant viral vectors into the brain might offer a therapeutic alternative, and is also under investigation for metachromatic leukodystrophy.

email