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Gene therapy for Parkinson's disease: contemporary practice and emerging concepts
- Source :
- Expert review of neurotherapeutics. 20(6)
- Publication Year :
- 2020
-
Abstract
- Gene therapy is a rapidly evolving technology that has predominantly utilized viral vectors to effectively deliver genetic material inside neurons to modulate the expression of one or more particular genes. Several gene therapy clinical trials have been conducted in Parkinson's disease (PD) by exploring strategies to either restore dopamine synthesis, enhance the production of trophic factors, enhance lysosomal function, or modify the interaction between different functional nodes of the basal ganglia.In this review, the authors sought to discuss contemporary practice, emerging concepts, and unmet needs for the future of gene therapy in PD.While safety has been demonstrated, clinical trials on gene therapy for PD highlight the need for higher than anticipated volumes of infusion in order to optimize dose and vector coverage. Neurosurgical delivery techniques for gene therapy have rapidly evolved from the use of multiple trans-frontal trajectories to a single parietooccipital shape-conforming infusion. The employment of convection-enhanced delivery with reflux-resistant cannulas has further improved the vector diffusion into the target structures. Future technological developments will reduce the invasiveness and duration of surgery, improve specificity and transduction capacity with novel capsid designs, and implement strategies to control transgene expression.
- Subjects :
- Parkinson's disease
biology
General Neuroscience
Genetic enhancement
Parkinson Disease
Genetic Therapy
biology.organism_classification
medicine.disease
medicine.disease_cause
Virology
030227 psychiatry
Viral vector
03 medical and health sciences
0302 clinical medicine
Lentivirus
medicine
Glial cell line-derived neurotrophic factor
biology.protein
Humans
Pharmacology (medical)
Neurology (clinical)
Vector (molecular biology)
Adeno-associated virus
030217 neurology & neurosurgery
Subjects
Details
- ISSN :
- 17448360
- Volume :
- 20
- Issue :
- 6
- Database :
- OpenAIRE
- Journal :
- Expert review of neurotherapeutics
- Accession number :
- edsair.doi.dedup.....aca6e3d4032dff99aca7a7ff9d604a7d