Single-dose AAV9-CLN6 gene transfer stabilizes motor and...

Molecular Genetics and Metabolism 2020 / 02 Vol. 129; Iss. 2

Single-dose AAV9-CLN6 gene transfer stabilizes motor and language function in CLN6-type Batten disease: Interim results from the first clinical gene therapy trial

de los Reyes, Emily, Meyer, Kathrin, Lehwald, Lenora, Albright, Charles, Castelli, Jeff, Jiang, Hai, Reha, Allen, Barth, Jay
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Volume:
129
Journal:
Molecular Genetics and Metabolism
DOI:
10.1016/j.ymgme.2019.11.101
Date:
February, 2020
File:
PDF, 346 KB
2020
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