Work

EMPOWERS: A phase 1/2 clinical trial of SB-318 ZFN-mediated in vivo human genome editing for treatment of MPS I (Hurler syndrome)

Year: 2019

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2018.12.163

Authors: Paul Harmatz, Heather A. Lau, Coy Heldermon, Nancy Leslie, Cheryl Wong Po Foo, Sagar A. Vaidya, Chester B. Whitley

Institutions: University of California, San Francisco, UCSF Benioff Children's Hospital, NYU Langone Health, University of Florida, Cincinnati Children's Hospital Medical Center, Sangamo BioSciences (United States), University of Minnesota

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: W2977606142

FWCI: 2.565

Cites:

Cited by: 21

Related to: 10