Articolo in rivista, 2021, CPE, 10.3390/ijms22094596

Ipscs: A preclinical drug research tool for neurological disorders

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;

Consiglio Nazionale delle Ricerche; Università degli Studi di Catania; Ortho-Neuro Center HUMANITAS Istituto Clinico Catanese

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).

International journal of molecular sciences (Print) 22 (9)

Keywords

AD, ALS, Drug development, FRAX, IPSCs, PD

CNR authors

Iemmolo Rosario, Bonaventura Gabriele, Cavallaro Sebastiano, La Cognata Valentina

CNR institutes

IRIB – Istituto per la Ricerca e l'Innovazione Biomedica