Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects.
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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.