Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.
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ABSTRACT:
SUBMITTER: Critchley BJ
PROVIDER: S-EPMC10014236 | biostudies-literature | 2023 Mar
REPOSITORIES: biostudies-literature
Publications
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.
Critchley Bethan J BJ Gaspar H Bobby HB Benedetti Sara S
Molecular therapy : the journal of the American Society of Gene Therapy 20221130 3
Lysosomal storage diseases (LSDs) are multisystem inherited metabolic disorders caused by dysfunctional lysosomal activity, resulting in the accumulation of undegraded macromolecules in a variety of organs/tissues, including the central nervous system (CNS). Treatments include enzyme replacement therapy, stem/progenitor cell transplantation, and in vivo gene therapy. However, these treatments are not fully effective in treating the CNS as neither enzymes, stem cells, nor viral vectors efficientl ...[more]
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