Co-expression of MGMTP140K and α-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correction
Author:
Publisher
Wiley
Subject
Genetics (clinical),Drug Discovery,Genetics,Molecular Biology,Molecular Medicine
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1. New function of a well‐known promoter: Enhancer activity of minimal CMV promoter enables efficient dual‐cassette transgene expression;The Journal of Gene Medicine;2021-08-18
2. miR-143 Regulates Lysosomal Enzyme Transport across the Blood-Brain Barrier and Transforms CNS Treatment for Mucopolysaccharidosis Type I;Molecular Therapy;2020-10
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5. Normalization and Improvement of CNS Deficits in Mice With Hurler Syndrome After Long-term Peripheral Delivery of BBB-targeted Iduronidase;Molecular Therapy;2014-12
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