Human gene therapy approaches for the treatment of Parkinson's disease: An overview of current and completed clinical trials.

@article{Hitti2019HumanGT,
  title={Human gene therapy approaches for the treatment of Parkinson's disease: An overview of current and completed clinical trials.},
  author={Frederick L. Hitti and Andrew Il Yang and Pedro Gonzalez-Alegre and Gordon Baltuch},
  journal={Parkinsonism \& related disorders},
  year={2019}
}
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28 Citations
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28 Citations

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Gene Therapy in the Management of Parkinson's Disease: Potential of GDNF as a Promising Therapeutic Strategy.
TLDR
The review primarily elaborates the significant role of glial cell-line derived neurotrophic factor in alleviating motor symptoms and loss of dopaminergic neurons in Parkinson's disease and establishes delivery aspects, like ventricular delivery of recombinant GDNF, intraparenchymal and intraputaminal delivery using infusion catheters.
Growth Factor Therapy for Parkinson’s Disease: Alternative Delivery Systems
TLDR
This review will give a short overview of some of the alternative delivery systems, with a focus on ex vivo gene therapy and biomaterial-aided protein and gene delivery, and will provide some perspectives on their potential for clinical development and translation.
Gene Therapeutic Approaches for the Treatment of Mitochondrial Dysfunction in Parkinson’s Disease
TLDR
This narrative review will discuss the experimental evidence suggesting mitochondrial dysfunction as a viable treatment target in patients with monogenic and idiopathic Parkinson’s disease and suggest mitochondria-targeted gene therapies are a potential strategy to improve an important primary disease mechanism in Parkinsonian disorders.
Strategies for the Treatment of Parkinson’s Disease: Beyond Dopamine
TLDR
The development of more reliable biomarkers at asymptomatic stages of the disease, and the use of genetic profiling of patients will surely permit a more effective treatment of PD.
Emerging Targeted Therapeutics for Genetic Subtypes of Parkinsonism
TLDR
The current obstacles towards the development of precision interventions in Parkinson’s disease are reviewed and the clinical trials that target genetic forms of PD, i.e., GBA-associated and LRRK2-associated PD are discussed.
Comparative efficacy of surgical approaches to disease modification in Parkinson disease
TLDR
Data underlying surgical approaches proposed to impart disease modification in PD are compared, showing that though targeted to engage potential mechanisms of PD these surgical approaches remain experimental, indicating the difficulty in translating therapeutic concepts into clinical practice.
Understanding the Potential of Genome Editing in Parkinson’s Disease
TLDR
The main biological pathways that become disrupted in Parkinson’s disease and their potential as gene therapy targets are highlighted and a comprehensive review of novel delivery vehicles available for gene-editing applications is performed.
Targeting Age-Related Neurodegenerative Diseases by AAV-Mediated Gene Therapy.
  • U. Cagin
  • Biology
    Advances in experimental medicine and biology
  • 2021
TLDR
The advantages of AAV-based gene therapies are discussed with emphasis on efforts of developing novel capsids with superior therapeutic efficacy and the results of clinical trials on AD, PD, and ALS are summarized.
Precision medicine in Parkinson’s disease: emerging treatments for genetic Parkinson’s disease
TLDR
Clinical trials that target genetic forms of Parkinson’s disease, i.e., GBA -associated and LRRK2 -associated PD, are reviewed, finding six ongoing studies which explicitely recruit GBA-PD patients, and two studies which recruit LRRk2 -PD patients.
Restoring Function to Dopaminergic Neurons: Progress in the Development of Cell-Based Therapies for Parkinson’s Disease
TLDR
The potential for cell-based therapies in Parkinson’s disease to restore the function of dopaminergic neurons is focused on, previous attempts to harness such strategies are critically reviewed, potential benefits and predicted limitations are discussed, and how previous roadblocks may be overcome are addressed.
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