The base editing of phenylalanine hydroxylase enzyme in the liver to restore phenylalanine metabolism and prevent neurotoxic effects.
This hearing test is tuned to a patient’s hearing level and changes based on performance to accurately diagnose hearing ability
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
Small molecules that bind to alpha-synuclein deposition and can be used for PET imagining in patients with synucleinopathies
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.
Novel derivatives of oleic acid effectively inhibiting ferroptosis that could be used for treatment of Friedreich Ataxia, neurodegenerative diseases, and neuropsychiatric diseases.
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy.
Brain penetrating compounds as promising therapeutics for treatment of neurodegenerative disorders.
Two novel monoclonal antibodies were discovered that target pathological conformations of the tau protein. The antibodies were shown in vitro and in vivo to inhibit the spread of pathological tau protein aggregates, which is one of the defining characteristics of AD.