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Updated: Jul 6, 2026
10:57
Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons
Published on: July 13, 2015
Advances in gene therapy for movement disorders.
Hideki Mochizuki1, Toru Yasuda, M Maral Mouradian
1Research Institute for Diseases of Old Age, Juntendo University School of Medicine, 2-1-1 Hongo, Bunkyo-ku, Tokyo 113-8421, Japan.
Summary
Gene therapy shows promise for Parkinson's disease (PD) and Huntington's disease (HD). Clinical trials using adeno-associated viral vectors are underway, targeting neurotransmission and neuroprotection for these neurodegenerative disorders.
Area of Science:
- Neuroscience
- Genetics
- Molecular Biology
Background:
- Parkinson's disease (PD) and Huntington's disease (HD) are debilitating neurodegenerative disorders.
- Decades of preclinical research have paved the way for clinical gene therapy applications.
Purpose of the Study:
- To review the current status of preclinical and clinical gene therapy for PD and HD.
- To discuss gene delivery vehicles, candidate genes, and therapeutic strategies.
Main Methods:
- Utilizing recombinant adeno-associated viral vectors for gene delivery to the brain.
- Testing candidate genes like glutamic acid decarboxylase, aromatic l-amino acid decarboxylase, and neurturin in PD.
- Employing RNA interference to repress mutant allele expression in HD models.
Main Results:
- Gene therapy approaches are demonstrating acceptable risk/benefit ratios in clinical trials for PD.
- Preclinical studies show potential for gene therapy to modulate neurotransmission, provide trophic effects, or correct genetic defects.
- RNA interference has shown promise in mitigating pathology in HD mouse models.
Conclusions:
- Gene therapy represents a promising therapeutic avenue for PD and HD.
- Ongoing research continues to refine gene delivery and target specific genetic underpinnings of these diseases.
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