30 août 2023
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C. Neftel et al., « Traitements à siRNA : état des lieux et perspectives [siRNA therapeutics : Current options and perspectives] », Serveur académique Lausannois, ID : 10.53738/REVMED.2023.19.839.1538
Biomedical research and the pharmaceutical industry constantly explore new therapeutic strategies. A growing understanding of genetic diseases has enabled the development of «gene therapies». These can act either by inducing the expression of deficient genes or by silencing pathological ones. Therapies using small interfering RNA (siRNA) form a new class of treatments capable of targeting and specifically inhibiting a gene of interest. First by using lipid nanoparticles and then by engineering specific chemical modifications, progress has been made in delivering siRNA specifically into hepatocytes, representing a particular interest in the field of hepatology. The aim of this review article is to describe the mode of action of siRNA therapeutics, to review currently available treatments as well as their application, and to enumerate future treatment possibilities that are still under development.