AAV vectors trigger DNA damage response-dependent pro-inflammatory signalling in human iPSC-derived CNS models and mouse brain.

Since the late 90’s there has been an increasing trend in the number of clinical trials using AAV vectors to deliver transgenes 50 , thanks to initial promising results of intravenous gene therapies targeting the liver for various metabolic diseases 51 , a setting in which the required doses are relatively low and AAV has shown a safe immunogenicity profile 2 .

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