Lentiviral vectors and, to a lesser extent, AAV vectors also elicit oncogenicity concerns arising from chromosomal integration and insertional mutagenesis. AAV vectors are further limited by their 4.7-kilobase packaging capacity poor tissue selectivity risk of liver toxicity and immunogenicity, which eliminates the possibility of redosing. “One of the issues is just the biomass needed to produce, for example, AAV at the scale and in the doses needed for large indications,” says Akin Akinc, CEO of Aera Therapeutics, a company developing a protein-nanoparticle-based delivery system. But their widespread deployment in dozens of clinical trials has exposed their limitations. Virus-based vectors - particularly those based on adeno-associated virus (AAV) and lentiviruses - have dominated the first wave of gene therapies to gain regulatory approval.
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