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Considerations for Preclinical Safety Assessment of Adeno-Associated Virus Gene Therapy Products

7 Oct 2018

Abstract excerpt

Progress in understanding the molecular bases of human health and disease in recent decades has flourished making it possible for the field of gene therapy (GT) to offer new possibilities for treating, and even curing, a plethora of medical conditions such as monogenic disorders and metabolic diseases. GT is a therapeutic intervention to genetically alter or modify living cells by means of gene delivery achieved...

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PMID: 30295175
DOI: 10.1177/0192623318803867

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