Work

AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies

Year: 2021

Type: article

Source: Trends in Molecular Medicine

DOI: 10.1016/j.molmed.2021.02.004

Authors: Huiya Yang, Robert H. Brown, Dan Wang, Kevin A. Strauss, Guangping Gao

Institutions: University of Massachusetts Chan Medical School, Clinic for Special Children

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: green

Funders: University of Massachusetts Medical School, National Heart, Lung, and Blood Institute, National Institute of Allergy and Infectious Diseases, National Institute of Neurological Disorders and Stroke

Awards: New Approaches to Gene Therapy for Alpha-1 Antitrypsin Deficiency, Next Generation of Recombinant AAV Serotype For Gene Therapy, An AAV-mediated functional cure and its impact on the reservoir, Develop combinatorial non-viral and viral CRISPR delivery for lung diseases, Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and Leukodystrophies

OpenAlex ID: W3133647047

FWCI: 1.129

Cites: 15

Cited by: 8

Related to: 10