For viral vector-based gene therapy, the transgene is packaged into a viral capsid, thus facilitating the delivery into the target cells. Most commonly, viral vectors used in inner ear gene therapy are based on adenoviruses, adeno-associated viruses (AAVs), or lentiviruses (LVs). These vectors...
(phase III; NCT03754790) for the treatment of hemophilia, iv) inclisiran (phase III; NCT03814187) for the treatment of hypercholesterolemia and, v) cemdisiran (phase II; NCT03999840) for the treatment of atypical hemolytic uremic syndrome. ALN-GO1, also called lumasiran, is a siRNA-...
TTV was present at a significantly higher rate in hemophiliacs (73%) and at lower rates in patients with cirrhosis (30%) and rheumatoid arthritis (28%). Patients with other liver diseases, systemic lupus erythematosus, or psoriasis or receiving hemodialysis had rates of infection similar to ...
Emicizumab in tolerized patients with hemophilia A with inhibitors: A single-institution pediatric cohort assessing inhibitor status. Res. Pract. Thromb. Haemost. 2021, 5, 342–348. [Google Scholar] [CrossRef] Pujhari, S.; Paul, S.; Ahluwalia, J.; Rasgon, J.L. Clotting disorder in ...