In my coursework, I am studying about viral vectors like adenoviruses and lentiviruses that are used for gene therapy. I understand that safety is a major concern, especially regarding insertional mutagenesis, immune reactions, or uncontrolled replication. Could someone explain what methods or strategies are generally followed to make sure these engineered vectors remain safe for clinical applications?
Kothapalli Shalini In genral;
there are standard methods to evaluate safety / toxicity / cytotoxicity of and pharmaceutical or biological compound or molecule. These are strictly required in order to obtain the necessary permissions / licences to manufacture the product and launch it to the market (based on FDA regulations).
In particular (regarding the specific nature of your question);
Researchers ensure the safety of engineered viral vectors for gene therapy by modifying viruses to be incapable of replicating or causing disease, removing viral genes that could cause harm, and using separate plasmids for different genetic elements to prevent the formation of replication-competent viruses. They also select specific viruses based on their ability to target cells, assess and minimize potential immune responses and off-target effects, and subject the vectors to rigorous testing and clinical trials to confirm safety and efficacy before human use.
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