Recent Advances in the Treatment of Genetic Forms of Parkinson's Disease: Hype or Hope?
DJ1
GBA
LRRK2
PINK1
PRKN
Parkinson’s disease
SNCA
genetic
treatment
Journal
Cells
ISSN: 2073-4409
Titre abrégé: Cells
Pays: Switzerland
ID NLM: 101600052
Informations de publication
Date de publication:
27 02 2023
27 02 2023
Historique:
received:
29
01
2023
revised:
22
02
2023
accepted:
24
02
2023
entrez:
11
3
2023
pubmed:
12
3
2023
medline:
15
3
2023
Statut:
epublish
Résumé
Parkinson's disease (PD) is a multifarious neurodegenerative disease. Its pathology is characterized by a prominent early death of dopaminergic neurons in the pars compacta of the substantia nigra and the presence of Lewy bodies with aggregated α-synuclein. Although the α-synuclein pathological aggregation and propagation, induced by several factors, is considered one of the most relevant hypotheses, PD pathogenesis is still a matter of debate. Indeed, environmental factors and genetic predisposition play an important role in PD. Mutations associated with a high risk for PD, usually called monogenic PD, underlie 5% to 10% of all PD cases. However, this percentage tends to increase over time because of the continuous identification of new genes associated with PD. The identification of genetic variants that can cause or increase the risk of PD has also given researchers the possibility to explore new personalized therapies. In this narrative review, we discuss the recent advances in the treatment of genetic forms of PD, focusing on different pathophysiologic aspects and ongoing clinical trials.
Identifiants
pubmed: 36899899
pii: cells12050764
doi: 10.3390/cells12050764
pmc: PMC10001341
pii:
doi:
Substances chimiques
alpha-Synuclein
0
Types de publication
Journal Article
Review
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
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