Restorative cell and gene therapies for Parkinson's disease
Barker, Roger A. LU and Björklund, Anders LU
(2023)
In Handbook of Clinical Neurology
193.
p.211-226
- Abstract
One of the core pathological features of Parkinson's disease (PD) is the loss of the dopaminergic nigrostriatal pathway which lies at the heart of many of the motor features of this condition as well as some of the cognitive problems. The importance of this pathological event is evident through the clinical benefits that are seen when patients with PD are treated with dopaminergic agents, at least in early-stage disease. However, these agents create problems of their own through stimulation of more intact dopaminergic networks within the central nervous system causing major neuropsychiatric problems including dopamine dysregulation. In addition, over time the nonphysiological stimulation of striatal dopamine receptors by L-dopa... (More)
One of the core pathological features of Parkinson's disease (PD) is the loss of the dopaminergic nigrostriatal pathway which lies at the heart of many of the motor features of this condition as well as some of the cognitive problems. The importance of this pathological event is evident through the clinical benefits that are seen when patients with PD are treated with dopaminergic agents, at least in early-stage disease. However, these agents create problems of their own through stimulation of more intact dopaminergic networks within the central nervous system causing major neuropsychiatric problems including dopamine dysregulation. In addition, over time the nonphysiological stimulation of striatal dopamine receptors by L-dopa containing drugs leads to the genesis of L-dopa-induced dyskinesias that can become very disabling in many cases. As such, there has been much interest in trying to better reconstitute the dopaminergic nigrostriatal pathway using either factors to regrow it, cells to replace it, or gene therapies to restore dopamine transmission in the striatum. In this chapter, we lay out the rationale, history and current status of these different therapies as well as highlighting where the field is heading and what new interventions might come to clinic in the coming years.
(Less)
- author
- Barker, Roger A.
LU
and Björklund, Anders
LU
- organization
- publishing date
- 2023
- type
- Chapter in Book/Report/Conference proceeding
- publication status
- published
- subject
- keywords
- Cell replacement therapies, Dopaminergic nigrostriatal pathway, GDNF, Gene therapies, In situ reprogramming, Neurturin, Parkinson's disease, Stem cells
- host publication
- Precision Medicine in Neurodegenerative Disorders, Part II
- series title
- Handbook of Clinical Neurology
- volume
- 193
- pages
- 16 pages
- publisher
- Elsevier Science Publishers B.V.
- external identifiers
-
- scopus:85148403470
- pmid:36803812
- ISSN
- 0072-9752
- 2212-4152
- ISBN
- 978-0-323-85555-6
- DOI
- 10.1016/B978-0-323-85555-6.00012-6
- language
- English
- LU publication?
- yes
- id
- 7415182f-0cc7-4ae9-9dec-7635bbd91485
- date added to LUP
- 2023-03-08 08:33:30
- date last changed
- 2024-09-05 21:19:35
@inbook{7415182f-0cc7-4ae9-9dec-7635bbd91485,
abstract = {{<p>One of the core pathological features of Parkinson's disease (PD) is the loss of the dopaminergic nigrostriatal pathway which lies at the heart of many of the motor features of this condition as well as some of the cognitive problems. The importance of this pathological event is evident through the clinical benefits that are seen when patients with PD are treated with dopaminergic agents, at least in early-stage disease. However, these agents create problems of their own through stimulation of more intact dopaminergic networks within the central nervous system causing major neuropsychiatric problems including dopamine dysregulation. In addition, over time the nonphysiological stimulation of striatal dopamine receptors by L-dopa containing drugs leads to the genesis of L-dopa-induced dyskinesias that can become very disabling in many cases. As such, there has been much interest in trying to better reconstitute the dopaminergic nigrostriatal pathway using either factors to regrow it, cells to replace it, or gene therapies to restore dopamine transmission in the striatum. In this chapter, we lay out the rationale, history and current status of these different therapies as well as highlighting where the field is heading and what new interventions might come to clinic in the coming years.</p>}},
author = {{Barker, Roger A. and Björklund, Anders}},
booktitle = {{Precision Medicine in Neurodegenerative Disorders, Part II}},
isbn = {{978-0-323-85555-6}},
issn = {{0072-9752}},
keywords = {{Cell replacement therapies; Dopaminergic nigrostriatal pathway; GDNF; Gene therapies; In situ reprogramming; Neurturin; Parkinson's disease; Stem cells}},
language = {{eng}},
pages = {{211--226}},
publisher = {{Elsevier Science Publishers B.V.}},
series = {{Handbook of Clinical Neurology}},
title = {{Restorative cell and gene therapies for Parkinson's disease}},
url = {{http://dx.doi.org/10.1016/B978-0-323-85555-6.00012-6}},
doi = {{10.1016/B978-0-323-85555-6.00012-6}},
volume = {{193}},
year = {{2023}},
}