Antigen-specific immunotherapy with engineered cytotoxic T cells targeting misformed proteins to treat neurodegenerative and muscular diseases.
Intranasal delivery of dantrolene nanoparticles can improve motor neuron function, muscle strength, and movement coordination and can offer therapeutic benefits for treatment of Amyotrophic Lateral Sclerosis (ASL).
Intranasal nasal dantrolene nanoparticles alone, or in combination with lithium, can provide protection against depression and dementia with minimal or no side effects.
Intranasal delivering of dantrolene nanoparticles could potentially be used to inhibit memory loss and reduce amyloid plaques in patients with Alzheimer's disease.
A machine learning technique that identifies biomarkers from neuroimaging data, allowing for identification and treatment of populations vulnerable to neurodegenerative diseases.
Peptide-functionalized lipid nanoparticles (pLNPs) for targeted messenger RNA (mRNA) delivery to the brain.
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