Work

Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.B

Year: 2018

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2017.12.075

Authors: Cristin Davidson, Alana Gibson, Tansy Gu, Randy Chandler, Benjamin Deverman, Viviana Gradinaru, Charles Venditti, William Pavan

Institutions: Albert Einstein College of Medicine, National Human Genome Research Institute, California Institute of Technology

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2792040440

FWCI:

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