iPSCs: A Preclinical Drug Research Tool for Neurological Disorders
The development and commercialization of new drugs is an articulated, lengthy, and very expensive process that proceeds through several steps, starting from target identification, screening new leading compounds for testing in preclinical studies, and subsequently in clinical trials to reach the fin...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-04-01
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| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/22/9/4596 |
| _version_ | 1797536133975900160 |
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| author | Gabriele Bonaventura Rosario Iemmolo Giuseppe Antonino Attaguile Valentina La Cognata Brigida Sabrina Pistone Giuseppe Raudino Velia D’Agata Giuseppina Cantarella Maria Luisa Barcellona Sebastiano Cavallaro |
| author_facet | Gabriele Bonaventura Rosario Iemmolo Giuseppe Antonino Attaguile Valentina La Cognata Brigida Sabrina Pistone Giuseppe Raudino Velia D’Agata Giuseppina Cantarella Maria Luisa Barcellona Sebastiano Cavallaro |
| author_sort | Gabriele Bonaventura |
| collection | DOAJ |
| description | The development and commercialization of new drugs is an articulated, lengthy, and very expensive process that proceeds through several steps, starting from target identification, screening new leading compounds for testing in preclinical studies, and subsequently in clinical trials to reach the final approval for therapeutic use. Preclinical studies are usually performed using both cell cultures and animal models, although they do not completely resume the complexity of human diseases, in particular neurodegenerative conditions. To this regard, stem cells represent a powerful tool in all steps of drug discovery. The recent advancement in induced Pluripotent Stem Cells (iPSCs) technology has opened the possibility to obtain patient-specific disease models for drug screening and development. Here, we report the use of iPSCs as a disease model for drug development in the contest of neurological disorders, including Alzheimer’s (AD) and Parkinson’s disease (PD), Amyotrophic lateral Sclerosis (ALS), and Fragile X syndrome (FRAX). |
| first_indexed | 2024-03-10T11:55:20Z |
| format | Article |
| id | doaj.art-ba4420c4988f4e389f7a62fa446f1234 |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T11:55:20Z |
| publishDate | 2021-04-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-ba4420c4988f4e389f7a62fa446f12342023-11-21T17:25:05ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672021-04-01229459610.3390/ijms22094596iPSCs: A Preclinical Drug Research Tool for Neurological DisordersGabriele Bonaventura0Rosario Iemmolo1Giuseppe Antonino Attaguile2Valentina La Cognata3Brigida Sabrina Pistone4Giuseppe Raudino5Velia D’Agata6Giuseppina Cantarella7Maria Luisa Barcellona8Sebastiano Cavallaro9Institute for Biomedical Research and Innovation (IRIB), Italian National Research Council, 95126 Catania, ItalyInstitute for Biomedical Research and Innovation (IRIB), Italian National Research Council, 95126 Catania, ItalyDepartment of Biomedical and Biotechnological Sciences (BIOMETEC), Section of Pharmacology, University of Catania, 95123 Catania, ItalyInstitute for Biomedical Research and Innovation (IRIB), Italian National Research Council, 95126 Catania, ItalyInstitute for Biomedical Research and Innovation (IRIB), Italian National Research Council, 95126 Catania, ItalyOrtho-Neuro Center HUMANITAS Istituto Clinico Catanese, 95045 Catania, ItalyDepartment of Biomedical and Biotechnological Sciences, Section of Anatomy, Histology and Movement Sciences, University of Catania, 95123 Catania, ItalyDepartment of Biomedical and Biotechnological Sciences (BIOMETEC), Section of Pharmacology, University of Catania, 95123 Catania, ItalyDepartment of Pharmaceutical Science, Biochemistry Section, University of Catania, 95123 Catania, ItalyInstitute for Biomedical Research and Innovation (IRIB), Italian National Research Council, 95126 Catania, ItalyThe development and commercialization of new drugs is an articulated, lengthy, and very expensive process that proceeds through several steps, starting from target identification, screening new leading compounds for testing in preclinical studies, and subsequently in clinical trials to reach the final approval for therapeutic use. Preclinical studies are usually performed using both cell cultures and animal models, although they do not completely resume the complexity of human diseases, in particular neurodegenerative conditions. To this regard, stem cells represent a powerful tool in all steps of drug discovery. The recent advancement in induced Pluripotent Stem Cells (iPSCs) technology has opened the possibility to obtain patient-specific disease models for drug screening and development. Here, we report the use of iPSCs as a disease model for drug development in the contest of neurological disorders, including Alzheimer’s (AD) and Parkinson’s disease (PD), Amyotrophic lateral Sclerosis (ALS), and Fragile X syndrome (FRAX).https://www.mdpi.com/1422-0067/22/9/4596iPSCsdrug developmentADALSPDFRAX |
| spellingShingle | Gabriele Bonaventura Rosario Iemmolo Giuseppe Antonino Attaguile Valentina La Cognata Brigida Sabrina Pistone Giuseppe Raudino Velia D’Agata Giuseppina Cantarella Maria Luisa Barcellona Sebastiano Cavallaro iPSCs: A Preclinical Drug Research Tool for Neurological Disorders International Journal of Molecular Sciences iPSCs drug development AD ALS PD FRAX |
| title | iPSCs: A Preclinical Drug Research Tool for Neurological Disorders |
| title_full | iPSCs: A Preclinical Drug Research Tool for Neurological Disorders |
| title_fullStr | iPSCs: A Preclinical Drug Research Tool for Neurological Disorders |
| title_full_unstemmed | iPSCs: A Preclinical Drug Research Tool for Neurological Disorders |
| title_short | iPSCs: A Preclinical Drug Research Tool for Neurological Disorders |
| title_sort | ipscs a preclinical drug research tool for neurological disorders |
| topic | iPSCs drug development AD ALS PD FRAX |
| url | https://www.mdpi.com/1422-0067/22/9/4596 |
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