Work

Single intravenous dose of AAVHSC15 vector with human phenylalanine hydroxylase transgene results in sustained correction of phenylketonuria in the PAH mouse model

Year: 2019

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2018.12.129

Authors: Omar L. Francone, Seemin S. Ahmed, Jeff L. Ellsworth, Deiby Faulkner, Arnold Sengooba, Hillard Rubin, Serena Dollive, Diana Lamppu, Teresa Wright, Albert Seymour

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2913416668

FWCI:

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