Work

Lenti in red: progress in gene therapy for human hemoglobinopathies

Year: 2004

Type: article

Abstract: Hemoglobinopathies are caused by abnormal structure or synthesis of hemoglobin chains and represent serious monogenic disorders. A new study demonstrates that lentiviral vectors can express clinically relevant levels of human transgenic beta-globin in red cells of xenografted mice. While some safety concerns must be addressed, this study is an important step toward potential clinical trials of gene therapy for hemoglobinopathies.

Source: Journal of Clinical Investigation

DOI: 10.1172/jci200423132

Authors: Christof von Kalle, Christopher Baum, David A. Williams

Institutions: Medizinische Hochschule Hannover, University of Cincinnati, Cincinnati Children's Hospital Medical Center

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: bronze

Funder: Cincinnati Children's Hospital Medical Center