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Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier |
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| Affiliation: | 1. Infection, Immunity and Inflammation Research & Teaching Department, UCL Great Ormond Street Institute of Child Health, Zayed Centre for Research, London WC1N 1DZ, UK;2. Orchard Therapeutics Ltd., London EC4N 6EU, UK;3. NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK |
| Abstract: |  |
| Keywords: | lysosomal storage diseases LSDs central nervous system CNS blood-brain barrier BBB enzyme replacement therapy ERT hematopoietic stem/progenitor cell transplantation HSC gene therapy |
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