Recombinant DNA Advisory Committee Updates Recommendations on Gene Transfer for X-Linked Severe Combined Immunodeficiency
Year: 2009
Type: article
Abstract: The National Institutes of Health Recombinant DNA Advisory Committee (RAC) has recently reviewed and amended its long-standing recommendations on gene transfer for X-linked severe combined immunodeficiency (X-SCID). The current recommendations have been in effect since shortly after the initial reports of serious adverse events that occurred in the human gene transfer studies to correct X-SCID in France (and a subsequent event in England). Faced with two new protocols submitted for review in its December 2008 and March 2009 meetings, the committee re-evaluated these recommendations, which essentially limited gene transfer trials in this disease to so-called salvage situations, i.e., to patients in whom identical or haplo-identical stem cell transplantation had failed.
Source: Molecular Therapy
DOI: 10.1038/mt.2009.76
Author: David A Williams
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2083021719