Work

Generation of β-Glucocerebrosidase variants with increased half-life in human plasma for liver directed AAV gene therapy aimed at the treatment of Gaucher disease type 1

Year: 2021

Type: article

Source: Molecular Genetics and Metabolism

DOI: 10.1016/j.ymgme.2020.12.047

Authors: Fabrizio Comper, I-Mei Yu, Petya Kalcheva, Carlos J. Miranda, Elisa Chisari, Clement Cocita, Samantha Correia, Jalpa Pandya, Natalie Northcott, Emmaline Stotter, Erald Shehu, Rose Sheridan, Amit C. Nathwani, Romuald Corbau

Institution: University College London

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Genetics (clinical)

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W3127048444

FWCI: 0.202

Cites:

Cited by: 2

Related to: 10