Work

240. Stable and Clinically Benign Clonal Dominance in an ADA-SCID Patient Treated With Retroviral Gene Therapy

Year: 2015

Type: article

Abstract: To date, adverse events involving genotoxicity and oncogenic transformation have occurred in several gene therapy trials using oncoretroviral vectors. In striking and surprising contrast, insertional oncogenesis has not been observed in multiple trials treating SCID caused by adenosine deaminase deficiency (ADA-SCID), despite the use of very similar oncoretroviral vectors. In our trial for ADA-SCID, we employed non-restrictive LAM-PCR to monitor clonality and observed that one patient developed a marked clonal dominance that was stable over multiple years.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33845-x

Authors: Aaron R. Cooper, Kit L. Shaw, Georgia R. Lill, Robert Sokolic, Fabio Candotti, Donald B. Kohn

Institutions: University of California, Los Angeles, National Institutes of Health, National Human Genome Research Institute

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