Work

FIXing hemophilia B with gene therapy

Year: 2006

Type: article

Abstract: A critical element for treatment of hemophilia B via gene therapy is sustained transgene expression. This study describes novel AAV vectors, optimized for expression in the liver, that generate sustained, therapeutic levels of factor IX expression in nonhuman primates. Although sustained

Source: Blood

DOI: 10.1182/blood-2006-01-0249

Author: John A. Chiorini

Institution: National Institute of Dental and Craniofacial Research

Language: English

Topic: Hemophilia Treatment and Research

Subfield: Hematology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: bronze

OpenAlex ID: W2070807533