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Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
- Published in:
- Molecular Therapy, 2015, v. 23, n. 8, p. 1298, doi. 10.1038/mt.2015.99
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- Article
Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I.
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- Molecular Therapy, 2014, v. 22, n. 12, p. 2018, doi. 10.1038/mt.2014.135
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- Article
Pharmacokinetics and distribution of 2‐hydroxypropyl‐β‐cyclodextrin following a single intrathecal dose to cats.
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- Journal of Inherited Metabolic Disease, 2020, v. 43, n. 3, p. 618, doi. 10.1002/jimd.12189
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- Article
Large animal models contribute to the development of therapies for central and peripheral nervous system dysfunction in patients with lysosomal storage diseases.
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- Human Molecular Genetics, 2019, v. 28, p. R119, doi. 10.1093/hmg/ddz127
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- Article