Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter Diseases Article Swipe
Joanne Ng
,
Serena Barral
,
Simon N. Waddington
,
Manju A. Kurian
·
YOU?
·
· 2023
· Open Access
·
· DOI: https://doi.org/10.1002/mds.29416
YOU?
·
· 2023
· Open Access
·
· DOI: https://doi.org/10.1002/mds.29416
Neurological disorders encompass a broad range of neurodegenerative and neurodevelopmental diseases that are complex and almost universally without disease modifying treatments. There is, therefore, significant unmet clinical need to develop novel therapeutic strategies for these patients. Viral gene therapies are a promising approach, where gene delivery is achieved through viral vectors such as adeno‐associated virus and lentivirus. The clinical efficacy of such gene therapies has already been observed in two neurological disorders of pediatric onset; for spinal muscular atrophy and aromatic L‐amino acid decarboxylase (AADC) deficiency, gene therapy has significantly modified the natural history of disease in these life‐limiting neurological disorders. Here, we review recent advances in gene therapy, focused on the targeted delivery of dopaminergic genes for Parkinson's disease and the primary neurotransmitter disorders, AADC deficiency and dopamine transporter deficiency syndrome (DTDS). Although recent European Medicines Agency and Medicines and Healthcare products Regulatory Agency approval of Upstaza (eladocagene exuparvovec) signifies an important landmark, numerous challenges remain. Future research will need to focus on defining the optimal therapeutic window for clinical intervention, better understanding of the duration of therapeutic efficacy, and improved brain targeting. © 2023 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
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Metadata
- Type
- review
- Language
- en
- Landing Page
- https://doi.org/10.1002/mds.29416
- https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/mds.29416
- OA Status
- hybrid
- Cited By
- 17
- References
- 53
- Related Works
- 10
- OpenAlex ID
- https://openalex.org/W4372334074
All OpenAlex metadata
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https://openalex.org/W4372334074Canonical identifier for this work in OpenAlex
- DOI
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https://doi.org/10.1002/mds.29416Digital Object Identifier
- Title
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Gene Therapy for Dopamine Dyshomeostasis: From Parkinson's to Primary Neurotransmitter DiseasesWork title
- Type
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reviewOpenAlex work type
- Language
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enPrimary language
- Publication year
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2023Year of publication
- Publication date
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2023-05-05Full publication date if available
- Authors
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Joanne Ng, Serena Barral, Simon N. Waddington, Manju A. KurianList of authors in order
- Landing page
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https://doi.org/10.1002/mds.29416Publisher landing page
- PDF URL
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https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/mds.29416Direct link to full text PDF
- Open access
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YesWhether a free full text is available
- OA status
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hybridOpen access status per OpenAlex
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https://onlinelibrary.wiley.com/doi/pdfdirect/10.1002/mds.29416Direct OA link when available
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Genetic enhancement, Medicine, Disease, Dopamine, Parkinson's disease, Dopaminergic, Movement disorders, Levodopa, Neuroscience, Bioinformatics, Psychology, Internal medicine, Biology, Gene, GeneticsTop concepts (fields/topics) attached by OpenAlex
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17Total citation count in OpenAlex
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2025: 9, 2024: 8Per-year citation counts (last 5 years)
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10Other works algorithmically related by OpenAlex
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| corresponding_institution_ids | https://openalex.org/I2800129641, https://openalex.org/I45129253 |
| sustainable_development_goals[0].id | https://metadata.un.org/sdg/17 |
| sustainable_development_goals[0].score | 0.4099999964237213 |
| sustainable_development_goals[0].display_name | Partnerships for the goals |
| citation_normalized_percentile.value | 0.9663149 |
| citation_normalized_percentile.is_in_top_1_percent | False |
| citation_normalized_percentile.is_in_top_10_percent | True |