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Novartis confirms Zolgensma caused tumors in two children via gene therapy vector integration
Novartis confirms Zolgensma's viral vector caused tumors in two children via DNA integration - but the FDA label still calls the risk 'theoretical.' ...
To design safe clinical trials, it is crucial to better understand and predict gene editing outcomes in preclinical studies. Bence György and collaborators have shown that adeno-associated viruses ...
A variety of mutations in lentiviral vector expression systems have been shown to generate a non-integrating phenotype. We studied a novel 12 base-pair U3-long terminal repeats (LTR) integrase (IN) ...
Adeno-associated virus (AAV) vectors are currently being used in several clinical gene-therapy trials (see the NIH OBA Human Gene Transfer Clinical Trials Database); however, little is known about the ...
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