Novel dual promoter AAV gene therapy platform ensures production of therapeutic soluble lysosomal enzymes with high M6P content to enable broad cellular uptake and cross correction

Authors :: Andrew Hedman
Jennifer Srnak
Yicheng Zhao
Shou Liu
Russell Gotshall
Lin Liu
Hung Do
Source :: Molecular Genetics and Metabolism. 138:107143
Publication Year :: 2023
Publisher :: Elsevier BV, 2023.

Subjects :: Endocrinology
Endocrinology, Diabetes and Metabolism
Genetics
Molecular Biology
Biochemistry

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