Gene Therapy Advances But Struggles to Interpret Safety Data in Small Animal Models
Year: 2006
Type: article
Abstract: Two recent studies will generate additional controversy in the therapeutic application of gene transfer to treat human diseases. Although these studies point to incremental and important advances, they highlight continued uncertainty about the relevance of studies in murine species with respect to human applications. In the first study, Manuel Grez and colleagues (Ott et al., Nat. Med., 2006) report the successful use of a gamma retrovirus vector to treat chronic granulomatous disease (CGD). CGD is caused by mutations in genes encoding proteins of the NADPH oxidase pathway.
Source: Molecular Therapy
Author: David A. Williams
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W1533323463