Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications
The pharmaceutical industry’s development of therapeutic medications for the treatment of Parkinson’s disease (PD) endures, as a result of the continuing need for better agents, and the increased clinical demand due to the aging population. Each new drug offers advantages and disadvantages to patien...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2012-06-01
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| Series: | Pharmaceuticals |
| Subjects: | |
| Online Access: | http://www.mdpi.com/1424-8247/5/6/553 |
| _version_ | 1818239124008599552 |
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| author | Massimo S. Fiandaca Howard J. Federoff Krystof S. Bankiewicz |
| author_facet | Massimo S. Fiandaca Howard J. Federoff Krystof S. Bankiewicz |
| author_sort | Massimo S. Fiandaca |
| collection | DOAJ |
| description | The pharmaceutical industry’s development of therapeutic medications for the treatment of Parkinson’s disease (PD) endures, as a result of the continuing need for better agents, and the increased clinical demand due to the aging population. Each new drug offers advantages and disadvantages to patients when compared to other medical offerings or surgical options. Deep brain stimulation (DBS) has become a standard surgical remedy for the effective treatment of select patients with PD, for whom most drug regimens have failed or become refractory. Similar to DBS as a surgical option, gene therapy for the treatment of PD is evolving as a future option. In the four different PD gene therapy approaches that have reached clinical trials investigators have documented an excellent safety profile associated with the stereotactic delivery, viral vectors and doses utilized, and transgenes expressed. In this article, we review the clinically relevant gene therapy strategies for the treatment of PD, concentrating on the published preclinical and clinical results, and the likely mechanisms involved. Based on these presentations, we advance an analysis of how the nature of the gene therapy used may eventually expand the scope and utility for the management of PD. |
| first_indexed | 2024-12-12T12:52:33Z |
| format | Article |
| id | doaj.art-0f4837bbf88e4b84b22819f11aca29b6 |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-12-12T12:52:33Z |
| publishDate | 2012-06-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-0f4837bbf88e4b84b22819f11aca29b62022-12-22T00:23:57ZengMDPI AGPharmaceuticals1424-82472012-06-015655359010.3390/ph5060553Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future IndicationsMassimo S. FiandacaHoward J. FederoffKrystof S. BankiewiczThe pharmaceutical industry’s development of therapeutic medications for the treatment of Parkinson’s disease (PD) endures, as a result of the continuing need for better agents, and the increased clinical demand due to the aging population. Each new drug offers advantages and disadvantages to patients when compared to other medical offerings or surgical options. Deep brain stimulation (DBS) has become a standard surgical remedy for the effective treatment of select patients with PD, for whom most drug regimens have failed or become refractory. Similar to DBS as a surgical option, gene therapy for the treatment of PD is evolving as a future option. In the four different PD gene therapy approaches that have reached clinical trials investigators have documented an excellent safety profile associated with the stereotactic delivery, viral vectors and doses utilized, and transgenes expressed. In this article, we review the clinically relevant gene therapy strategies for the treatment of PD, concentrating on the published preclinical and clinical results, and the likely mechanisms involved. Based on these presentations, we advance an analysis of how the nature of the gene therapy used may eventually expand the scope and utility for the management of PD.http://www.mdpi.com/1424-8247/5/6/553convection-enhanced deliveryenzyme-replacement therapygene therapyneurotrophic factorParkinson’s diseaseviral vector |
| spellingShingle | Massimo S. Fiandaca Howard J. Federoff Krystof S. Bankiewicz Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications Pharmaceuticals convection-enhanced delivery enzyme-replacement therapy gene therapy neurotrophic factor Parkinson’s disease viral vector |
| title | Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications |
| title_full | Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications |
| title_fullStr | Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications |
| title_full_unstemmed | Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications |
| title_short | Gene Therapy for the Treatment of Parkinson’s Disease: The Nature of the Biologics Expands the Future Indications |
| title_sort | gene therapy for the treatment of parkinson s disease the nature of the biologics expands the future indications |
| topic | convection-enhanced delivery enzyme-replacement therapy gene therapy neurotrophic factor Parkinson’s disease viral vector |
| url | http://www.mdpi.com/1424-8247/5/6/553 |
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