Work

Flushing Out Antibodies to Make AAV Gene Therapy Available to More Patients

Year: 2013

Type: article

Abstract: Six men with hemophilia recently underwent intravenous infusion of a vector based on adeno-associated virus (AAV) serotype 8 that expressed factor IX (FIX), which is deficient in such patients. Gene transfer led to levels of expression of FIX that were between 1% and 6% of that in normal individuals for more than two years, and to conversion from severe hemophilia B to mild or moderate disease.1,2 However, these patients had been carefully selected to exclude those with preexisting natural immunity against AAV in the form of neutralizing antibodies (NAbs).

Source: Molecular Therapy

DOI: 10.1038/mt.2013.5

Authors: Steven E Raper, James M Wilson, Frederick A Nunes

Institutions: University of Pennsylvania, Pennsylvania Hospital

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: hybrid

Estimated APC paid: $4,500