Targeting Root Cause by Systemic scAAV9-h Gene Delivery: Functional Correction and Reversal of Severe MPS II in Mice (Q58765137)

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Kim Zaraspe

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Naoko Murakami

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Aaron S Meadows

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Ricardo J Pineda

5

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Douglas M McCarty

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Joseph Muenzer

Molecular Therapy. Methods & Clinical Development

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language of work or name

English

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publication date

21 September 2018

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published in

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volume

10

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page(s)

327-340

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cites work

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12 December 2020

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12 December 2020

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Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating MPS IIIB: Toxicology, Bio-distribution and Immunological Assessments in Primates

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12 December 2020

A GLP-compliant toxicology and biodistribution study: systemic delivery of a rAAV9 vector for the treatment of mucopolysaccharidosis IIIB.

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12 December 2020