Towards next generation polymers for Gene-Delivery applications
JULIETTE MARTIN
Scientific Communication Manager at SEQENS Innovative BU – CRDO
Coronavirus disease 2019 (COVID-19) is the historical landmark for the expanding development of mRNA therapeutics. More specifically, nucleic acid-based therapies have emerged as promising and efficient approaches to treat diseases, including genetic disorders, cancers, neurological disorders. Indeed, nucleic acids can be used to correct a disease-related inherited gene by adding, removing, or replacing genetic material, or to make cells and tissues behave in a certain way by modifying gene expression (1). Importantly, nucleic acid-based therapeutics must act intracellularly, although most of them are rapidly cleared and degraded after systemic administration and do not really cross the plasma membrane.
As a result, the use of gene-delivery vectors to protect and enable their transport inside cells with therapeutic efficiency and with minimal adverse effects to patients, are essential. To date, gene-delivery vectors are mainly classified into two major categories, including viral and nonviral vectors. The use of viral vectors has raised considerable safety concerns, such as immune responses, inflammatory responses, toxicity problems, and insertional mutagenesis. Consequen ...