Small molecule therapeutics that aid in clearing abnormal protein deposits observed in neurodegenerative diseases.
Hydroxycholesterols are used to formulate lipid nanoparticles (LNPs) for enhanced mRNA delivery into T cells.
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.
Ten candidate serum and synovial protein levels provide early indication of joint impairment in lysosomal storage disorders.
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy.
Virtual reality-based system and software for quick, accurate, and reproducible evaluation of functional vision capacity following clinical treatment
Immobilized solid-phase DNA deamination for epigenetic sequencing of sparse DNA samples such as cell-free DNA.
Small molecules targeting utrophin mRNA untranslated regions overcome post-transcriptional repression