Parkinson's Disease: Gene Therapies

Prospects for polymer therapeutics in Parkinson's disease and other neurodegenerative disorders

“…However, non-viral vectors are not without their advantages, which could materialize in later years once the efficacy has been improved. If viral mediated gene therapy can pave the way to prove the principle that gene therapies can be of benefit to PD patients [230,231], then perhaps the advantages of nonviral vectors such as ease of handling/distribution/storage, and ability in most cases for large scale production could emerge allowing widespread therapeutic intervention. In addition, targeted vectors, with the ability to cross the BBB [232], would significantly reduce the cost of intervention.…”

Focused ultrasound-induced blood-brain barrier opening for non-viral, non-invasive, and targeted gene delivery

“…Recently, numerous preclinical and clinical studies have demonstrated the potential use of gene therapy for neurodegenerative diseases [2,3]. With the delivery of genes into the CNS, it may be possible to genetically modify neuronal cells that are directly functionally impaired, potentially relieving symptoms or even reversing the progression of the neurodegenerative diseases [4,5].…”

“…The BBB is formed by the tight junctions between the endothelial cells responsibly for the barrier function, preventing uptake of most therapeutic agents into the brain. To overcome this, current clinical trials have applied local gene delivery through direct injection [5,8], however, the craniotomy/burr-hole installation process prior to gene injection is highly invasive. When attempting non-invasive gene delivery through the circulation, there is the challenge of preventing the rapid degradation of naked DNA by nucleases in circulation and rapid clearance of DNA by the RES systems [9].…”

Evidence of mutations inRIC3acetylcholine receptor chaperone as a novel cause of autosomal-dominant Parkinson's disease with non-motor phenotypes

“…Gene(s) from other pathways including inflammatory, programmed cell death, dopaminergic, cholinergic and so on, which in principle could very well contribute to the monogenic forms of PD, have not yet been discovered. Interestingly, the known genes have provided some pharmacological leads20 but do not explain all familial forms of PD tested to date. With the advent of NGS which enables effective screening of all families with disease, irrespective of their size, the possibility of causal gene discovery seems enhanced.…”