Work

The treatment of biochemical genetic diseases: From substrate reduction to nucleic acid therapies

Year: 2023

Type: review

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2023.107693

Authors: E. Naomi Vos, Didem Demirbas, Matthew Mangel, M. Estela Rubio-Gozalbo, Harvey L. Levy, Gerard T. Berry

Institutions: Boston Children's Hospital, Harvard University, Manton Center for Orphan Disease Research, Maastricht University Medical Centre, Maastricht University, MetabERN

Language: English

Topic: Metabolism and Genetic Disorders

Subfield: Clinical Biochemistry

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4386295919

FWCI: 0.225

Cites: 157

Cited by: 6

Related to: 10