Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
A method of treating Fragile X syndrome by correcting the abnormal DNA clusters causing the characteristic symptoms.
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.
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy.
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac
Ten candidate serum and synovial protein levels provide early indication of joint impairment in lysosomal storage disorders.
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.
Virtual reality-based system and software for quick, accurate, and reproducible evaluation of functional vision capacity following clinical treatment
Small molecules targeting utrophin mRNA untranslated regions overcome post-transcriptional repression