Anastasia Khvorova

Apr 3, 2024 | nature.com | Qi Tang |Anastasia Khvorova

AbstractMore than 25 years after its discovery, the post-transcriptional gene regulation mechanism termed RNAi is now transforming pharmaceutical development, proved by the recent FDA approval of multiple small interfering RNA (siRNA) drugs that target the liver. Synthetic siRNAs that trigger RNAi have the potential to specifically silence virtually any therapeutic target with unprecedented potency and durability.

Nov 4, 2023 | nature.com | Qi Tang |Hassan Fakih |Vignesh Hariharan |Ashley Summers |Ken Okamura |Kathryn R. Monopoli | +14 more

AbstractInhibition of Janus kinase (JAK) family enzymes is a popular strategy for treating inflammatory and autoimmune skin diseases. In the clinic, small molecule JAK inhibitors show distinct efficacy and safety profiles, likely reflecting variable selectivity for JAK subtypes. Absolute JAK subtype selectivity has not yet been achieved.

Jun 12, 2023 | jamanetwork.com | Anastasia Khvorova

Small interfering RNA (siRNA) is one of several nucleic acid molecules representing a third class of therapeutic modalities, in addition to small molecules and biologics. Like small molecules, siRNAs have a defined chemical structure, are inexpensive to manufacture, and are stable at room temperature, but differ in their mechanism of action.