Work

615. Choroid Plexus-Targeted Viral Gene Therapy for Lysosomal Storage Diseases

Year: 2016

Type: article

Abstract: Lysosomal storage diseases (LSDs) represent a category of approximately 60 inherited neurometabolic illnesses with considerable morbidity and mortality in children and adults. The burden of LSD is high due to the chronic progressive decline in the neurocognitive function of affected individuals that profoundly limits their societal integration. Current therapy for LSDs has focused on recombinant enzyme replacement therapy (ERT), which while promising, is not ideal for numerous reasons, including the short half-lives of ERT with weekly or monthly administration, potential immune responses against ERT may also reduce the effectiveness for the treatment and inefficient passage through the blood-brain barrier.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33423-2

Authors: Eun-Young Choi, Shih-hsin Kan, S.Q. Le, Patricia I. Dickson, Stephen G. Kaler

Institutions: National Institutes of Health, The Lundquist Institute, Harbor–UCLA Medical Center

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500