Novel therapeutics for diverse neurodegenerative disorders

Abstract

Neurodegenerative diseases are a diverse category of illnesses that are characterized by the progressive degradation of the central nervous system and peripheral nervous system. Examples of novel therapeutics in NDDs include gene therapy, immunomodulation, liposomal-based therapy, neuromodulation, targeting protein aggregation and misfolding, using autophagy, using neurotrophic factors, and stem cell therapy. Gene therapy includes vector-based gene therapies and RNA-based gene therapies. Immuno-modulation strategies include therapeutics targeting inflammatory markers and passive or active immunization. Drug delivery by liposomal carriers can avoid obstacles in reaching the target in brain parenchyma like the blood-brain barrier. NDDs, essential tremors, epilepsy, dystonia, headaches, obsessive-compulsive disorder, and pain can all be treated with neuromodulation techniques. For many NDDs, preventing protein aggregation and misfolding is a potential therapeutic target. Inducing autophagy can be used in NDDs since it is a key intracellular mechanism for clearing aggregated proteins and damaged organelles. Neurotrophic factors like GDNF, brain-derived neurotrophic factor, and NGF can influence the growth, differentiation, and functioning of neurons. Stem cell therapy can replace degenerated neurons, control inflammation, decrease apoptosis, and stimulate the release of growth factors and neurotrophic factors. Therapeutic efforts should incorporate a variety of different techniques for effective therapy together with next-generation delivery devices. © 2024 Elsevier Inc. All rights reserved.