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Genetic therapy

Japanese researchers studied the effects of locally administered ASOs on mouse models of Parkinson's disease. Their research indicates that ASOs can help prevent the formation and spread of harmful alpha-synuclein aggregates in different brain regions, suggesting ASOs as a potential therapeutic strategy for preventing and controlling the progression of neurodegenerative diseases.

New Treatment Approach Shows Promise in Slowing Parkinson’s Disease Progression

A young patient and her parents

Researchers Develop Potential Genetic Therapy to Reverse Giant Moles in Rare Skin Condition

Edward Hopper style illustration of a woman checking her cholesterol in a sparse hotel room

Gene-editing treatment could replace cholesterol meds

Pathological phosphorylation (yellow) of Tau proteins (red-orange) leads to disintegration of microtubuli in the neuron axon an aggregation of the tau proteins. The transport of synaptic vesicles (orange-blue) is interrupted. Credit: selvanegra on iStock

First ‘gene silencing’ drug for Alzheimer’s disease shows promise

Liver illustration

Nanoparticle stops liver fibrosis in its tracks, reverses damage

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