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Article

Augmentation of transgene-encoded protein after neonatal injection of adeno-associated virus improves hepatic copy number without immune responses

Achieving persistent expression is a prerequisite for genetic therapies for inherited metabolic enzymopathies. Such disorders potentially could be treated with gene therapy shortly after birth to prevent patho...

Denise S. Tai, Chuhong Hu, Elizabeth H. Kim, Gerald S. Lipshutz in Pediatric Research (2015)