Year: 2009
Type: article
Abstract: Two recent reports of note for readers of Molecular Therapy—one in the scientific literature and one in the “regulatory cyperspace”—point to continued success and ongoing challenges of the gene therapy field. Pule et al. recently reported that they had successfully engineered virus-specific T cells that demonstrated apparent antitumor efficacy against neuroblastoma, a rare but deadly tumor seen in children.1 At the same time, many of us received a “Notification of Serious Adverse Event” from the National Institutes of Health Office of Biotechnology Activities that was based on a press release by the French Health Products Safety Agency (Agence Française de Sécurité Sanitaire des Produits de Santé; AFSSAPS) reporting “a biological observation in one treated patient” in a trial in France using a lentivirus (HIV-1) vector containing the gene for human β-globin to transduce CD34+ hematopoietic stem cells in patients with severe forms of thalassemia or sickle cell disease.
Source: Molecular Therapy
DOI: 10.1038/mt.2009.162
Author: David A Williams
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $4,500
OpenAlex ID: W2031906846