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Advexin, a similar gene therapy approach from Introgen, was turned down by the US Food and Drug Administration (FDA) in 2008. [4] Concerns about the safety of adenovirus vectors were raised after the 1999 death of Jesse Gelsinger while participating in a gene therapy trial. Since then, work using adenovirus vectors has focused on genetically ...
Several features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. [3] Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell.
Gene therapy seeks to modulate or otherwise affect gene expression via the introduction of a therapeutic transgene. Gene therapy by viral vectors can be performed by in vivo delivery by directly administering the vector to the patient, or ex vivo by extracting cells from the patient, transducing them, and then reintroducing the modified cells ...
The original issue focused on viral vectors for gene delivery vectors and use of gene therapy treatments for oncology. Current Gene Therapy was among the first gene therapy focused journals to publish on the use of Alphaviruses for gene therapy, [2] oncolytic virotherapy using adenoviral-mediated gene transfer, [3] and the use of Fas Ligand ...
Self-complementary adeno-associated virus (scAAV) is a viral vector engineered from the naturally occurring adeno-associated virus (AAV) to be used as a tool for gene therapy. [1] Use of recombinant AAV (rAAV) has been successful in clinical trials addressing a variety of diseases. [ 2 ]
Then, viral gene expression can occur, without integrating the viral genome into host cell chromosomes, [23] and new virus particles can be generated. The adenovirus life cycle is separated by the DNA replication process into two phases: an early and a late phase. [2]
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