Preclinical development of a platform for enzyme therapy in...

Molecular Genetics and Metabolism 2018 / 02 Vol. 123; Iss. 2

Preclinical development of a platform for enzyme therapy in the CNS of MPS I and MPS II patients based on intrathecal AAV delivery

Hinderer, Christian, Hordeaux, Juliette, Katz, Nathan, Bell, Peter, Goode, Tamara, Haskins, Mark E., Wilson, James M.
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Volume:
123
Language:
english
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2017.12.156
Date:
February, 2018
File:
PDF, 49 KB
english, 2018
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