Work

C-8. Immunological and Metabolic Correction After Lentiviral Vector Gene Therapy for ADA Deficiency

Year: 2015

Type: article

Abstract: Background: Adenosine deaminase deficiency leads to severe combined immunodeficiency. Autologous haematopoietic stem cell gene therapy may offer a curative therapy. We developed a self inactivating lentiviral vector in which the human ADA gene is driven by an internal EFS promoter. Parallel trials using this vector were conducted in UCL, London and UCLA, Los Angeles

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33866-7

Authors: Hubert B. Gaspar, Karen Buckland, Denise A. Carbonaro, Kit Shaw, Provobati Barman, Alejandra Davila, Kimberly C. Gilmour, Claire Booth, Dayna Terrazs, Kenneth Cornetta, Anna Paruzynski, Manfred Schmidt, Robert Sokolic, Fabio Candotti, Adrian J. Thrasher, Donald B. Kohn

Institutions: University College London, University of California, Los Angeles, Indiana University Indianapolis, Heidelberg University, National Institutes of Health

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

OpenAlex ID: W2561404733

FWCI: 1.826

Cites:

Cited by: 10

Related to: 10