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Targeting the Central Nervous System in Lysosomal Storage Diseases: Strategies to Deliver Therapeutics Across the Blood-Brain Barrier

Critchley, Bethan J; Gaspar, H Bobby; Benedetti, Sara; (2023) Targeting the Central Nervous System in Lysosomal Storage Diseases: Strategies to Deliver Therapeutics Across the Blood-Brain Barrier. Molecular Therapy , 31 (3) pp. 657-675. 10.1016/j.ymthe.2022.11.015. Green open access

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Abstract

Lysosomal storage diseases (LSDs) are multisystem inherited metabolic disorders caused by dysfunctional lysosomal enzymes, 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 efficiently cross the blood-brain barrier (BBB). Here we will review the latest advancements in improving delivery of different therapeutic agents to the CNS and comment upon outstanding questions in the field of neurological LSDs.

Type: Article
Title: Targeting the Central Nervous System in Lysosomal Storage Diseases: Strategies to Deliver Therapeutics Across the Blood-Brain Barrier
Location: United States
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.ymthe.2022.11.015
Publisher version: https://doi.org/10.1016/j.ymthe.2022.11.015
Language: English
Additional information: This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions.
Keywords: Lysosomal storage diseases (LSDs), blood-brain barrier (BBB), central nervous system (CNS), enzyme replacement therapy (ERT), gene therapy (GT), haematopoietic stem/progenitor cell (HSPC) transplantation
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept
URI: https://discovery.ucl.ac.uk/id/eprint/10161852
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