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The intersection of vector biology, gene therapy, and hemophilia

1 Aug 2021

Abstract excerpt

Gene therapy is at the forefront of the drive to bring the potential of cure to patients with genetic diseases. Multiple mechanisms of effective and efficient gene therapy delivery (eg, lentiviral, adeno-associated) for transgene expression as well as gene editing have been explored to improve vector and construct attributes and achieve therapeutic success. Recent clinical research has focused on recombinant...

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PMID: 34485808
DOI: 10.1002/rth2.12586

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