From lab bench to hope: a review of gene therapies in clinical trials for Parkinson’s disease and challenges

Grote, Julia; Patel, Nikita; Bates, Chad; Parmar, Mayur S.

doi:10.1007/s10072-024-07599-1

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2024

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Cited by 6 publications

References 56 publications

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From Lab Bench to Hope: Emerging Gene Therapies in Clinical Trials for Alzheimer’s Disease

Ortega,

Chernicki,

et al. 2024

Mol Neurobiol

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From Lab Bench to Hope: Emerging Gene Therapies in Clinical Trials for Alzheimer’s Disease

Ortega,

Chernicki,

et al. 2024

Mol Neurobiol

View full text Add to dashboard Cite

Novel approaches targeting α-Synuclein for Parkinson's Disease: Current progress and future directions for the disease-modifying therapies

Baggett,

Olson,

Parmar

2024

Brain Disorders

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Serotonin system development and adult function are regulated by GDNF

Garton,

Runneberger,

Casserly

et al. 2024

Preprint

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Cognitive functions, neuropsychiatric disorders and behaviors from feeding to mood relate to the serotonin (5-hydroxytryptamine; 5-HT) system. We report that expression of glial cell line-derived neurotrophic factor (GDNF), a known potent stimulator of the brain dopamine system, correlates with serotonergic markers in humans, and increased GDNF defines a subset of psychiatric patients with a characteristic 5-HT-related gene expression pattern. A similar ∼1.5- to 2-fold upregulation of endogenous GDNF expression in mice increases brain 5-HT levels and function, both developmentally and during adulthood, and modulates response to fluoxetine. Notably, increasing GDNF more than approximately 2-fold does not increase 5-HT further and instead produces an inverted U-shaped curve of 5-HT levels, suggesting why the GDNF/5-HT correlation has remained controversial. Collectively, our data indicate that GDNF levels fine-tune 5-HT system development and adult function while excess GDNF exclusively associates with neuropsychiatric illness, making it an important target for future research.

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From lab bench to hope: a review of gene therapies in clinical trials for Parkinson’s disease and challenges

Cited by 6 publications

References 56 publications

From Lab Bench to Hope: Emerging Gene Therapies in Clinical Trials for Alzheimer’s Disease

From Lab Bench to Hope: Emerging Gene Therapies in Clinical Trials for Alzheimer’s Disease

Novel approaches targeting α-Synuclein for Parkinson's Disease: Current progress and future directions for the disease-modifying therapies

Serotonin system development and adult function are regulated by GDNF

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