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http://dx.doi.org/10.1016/j.ymthe.2021.06.011 | DOI Listing |
Mol Ther
August 2022
Drug Safety Research and Development, Pfizer Inc., Cambridge, MA, USA.
On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in preclinical and clinical AAV gene therapy, to further contextualize and understand this phenomenon. Recombinant AAV (rAAV) vectors are used to develop therapies for many conditions given their ability to transduce multiple cell types, resulting in long-term expression of transgenes. Although most rAAV DNA typically remains episomal, some rAAV DNA becomes integrated into genomic DNA at a low frequency, and rAAV insertional mutagenesis has been shown to lead to tumorigenesis in neonatal mice.
View Article and Find Full Text PDFMol Ther
September 2021
Executive Director, Informa Pharma Custom Intelligence, New York, NY, USA.
Mol Ther
July 2021
Executive Editor of the Molecular Therapy family of journals, Molecular Therapy, Seattle, WA, USA.
JHEP Rep
August 2021
Gene Therapy Area, Foundation for Applied Medical Research, University of Navarra, IdisNA, Pamplona, Spain.
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