Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy
Understanding the pathophysiology and genetic background of Parkinson’s disease (PD) increases the likelihood of developing effective disease-modifying therapeutic strategies. In particular, the discovery of genetic variants causing or increasing the risk for PD has contributed to refining the clini...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-09-01
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| Series: | Brain Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2076-3425/12/10/1308 |
| _version_ | 1827651245429489664 |
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| author | Giulia Straccia Fabiana Colucci Roberto Eleopra Roberto Cilia |
| author_facet | Giulia Straccia Fabiana Colucci Roberto Eleopra Roberto Cilia |
| author_sort | Giulia Straccia |
| collection | DOAJ |
| description | Understanding the pathophysiology and genetic background of Parkinson’s disease (PD) increases the likelihood of developing effective disease-modifying therapeutic strategies. In particular, the discovery of genetic variants causing or increasing the risk for PD has contributed to refining the clinical, biological, and molecular classification of the disease and has offered new insights into sporadic forms. It is even more evident that specific genetic mutations can show different responses to pharmacological and device-aided therapies. To date, several agents acting on multiple PD-causing pathogenic pathways have been tested as disease-modifying strategies, with disappointing results. This may be caused by the recruitment of PD populations whose underlying molecular pathophysiology is heterogeneous. We believe that an effective model of personalized medicine must be prioritized in the near future. Here, we review the current therapeutic options under clinical and preclinical development for PD and discuss the key pending questions and challenges to face for successful clinical trials. Furthermore, we provide some insights into the role of genetics in guiding the decision-making process on symptomatic and device-aided therapies for PD in daily clinical practice. |
| first_indexed | 2024-03-09T20:35:01Z |
| format | Article |
| id | doaj.art-76b6457bee2c45baa9a0d0296d3de165 |
| institution | Directory Open Access Journal |
| issn | 2076-3425 |
| language | English |
| last_indexed | 2024-03-09T20:35:01Z |
| publishDate | 2022-09-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Brain Sciences |
| spelling | doaj.art-76b6457bee2c45baa9a0d0296d3de1652023-11-23T23:13:20ZengMDPI AGBrain Sciences2076-34252022-09-011210130810.3390/brainsci12101308Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized TherapyGiulia Straccia0Fabiana Colucci1Roberto Eleopra2Roberto Cilia3Neurology and Stroke Unit, Centro Traumatologico Ortopedico (C.T.O) Hospital, A.O.R.N., Ospedali dei Colli, 80131 Naples, ItalyFondazione IRCCS Istituto Neurologico Carlo Besta, Department of Clinical Neurosciences, Parkinson and Movement Disorders Unit, 20133 Milan, ItalyFondazione IRCCS Istituto Neurologico Carlo Besta, Department of Clinical Neurosciences, Parkinson and Movement Disorders Unit, 20133 Milan, ItalyFondazione IRCCS Istituto Neurologico Carlo Besta, Department of Clinical Neurosciences, Parkinson and Movement Disorders Unit, 20133 Milan, ItalyUnderstanding the pathophysiology and genetic background of Parkinson’s disease (PD) increases the likelihood of developing effective disease-modifying therapeutic strategies. In particular, the discovery of genetic variants causing or increasing the risk for PD has contributed to refining the clinical, biological, and molecular classification of the disease and has offered new insights into sporadic forms. It is even more evident that specific genetic mutations can show different responses to pharmacological and device-aided therapies. To date, several agents acting on multiple PD-causing pathogenic pathways have been tested as disease-modifying strategies, with disappointing results. This may be caused by the recruitment of PD populations whose underlying molecular pathophysiology is heterogeneous. We believe that an effective model of personalized medicine must be prioritized in the near future. Here, we review the current therapeutic options under clinical and preclinical development for PD and discuss the key pending questions and challenges to face for successful clinical trials. Furthermore, we provide some insights into the role of genetics in guiding the decision-making process on symptomatic and device-aided therapies for PD in daily clinical practice.https://www.mdpi.com/2076-3425/12/10/1308Parkinson’s diseaseprecision medicinedisease-modifying therapygeneticsGBA |
| spellingShingle | Giulia Straccia Fabiana Colucci Roberto Eleopra Roberto Cilia Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy Brain Sciences Parkinson’s disease precision medicine disease-modifying therapy genetics GBA |
| title | Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy |
| title_full | Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy |
| title_fullStr | Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy |
| title_full_unstemmed | Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy |
| title_short | Precision Medicine in Parkinson’s Disease: From Genetic Risk Signals to Personalized Therapy |
| title_sort | precision medicine in parkinson s disease from genetic risk signals to personalized therapy |
| topic | Parkinson’s disease precision medicine disease-modifying therapy genetics GBA |
| url | https://www.mdpi.com/2076-3425/12/10/1308 |
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