Work

Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia

Year: 1994

Type: article

Study design: Case Report

Source: Nature Genetics

DOI: 10.1038/ng0494-335

Authors: Mariann Grossman, Steven E. Raper, Karen Kozarsky, Evan A. Stein, John F. Engelhardt, David Muller, Paul J. Lupien, James M. Wilson

Institutions: University of Michigan, Michigan Medicine, Christ Hospital, Christ Hospital, Université Laval

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

Funders: National Institutes of Health, National Institute of Diabetes and Digestive and Kidney Diseases, National Center for Research Resources

Awards: LIVER-DIRECTED GENE THERAPIES, G213410136, G2519210435, G3165480743, EXPERIMENTAL MODELS OF GENE THERAPY, General Clinical Research Center

OpenAlex ID: W2166797660

FWCI: 37.7

Cites: 17

Cited by: 558

Related to: 10