iPSCs: A Preclinical Drug Research Tool for Neurological Disorders

• Published in: International journal of molecular sciences Vol. 22; no. 9; p. 4596
• Main Authors: Bonaventura, Gabriele, Iemmolo, Rosario, Attaguile, Giuseppe Antonino, La Cognata, Valentina, Pistone, Brigida Sabrina, Raudino, Giuseppe, D'Agata, Velia, Cantarella, Giuseppina, Barcellona, Maria Luisa, Cavallaro, Sebastiano
• Format: Journal Article
• Language: English
• Published: Switzerland MDPI AG 27-04-2021; MDPI
• Subjects: ALS; Alzheimer's disease; Amyotrophic lateral sclerosis; Animal models; Cell culture; Clinical trials; Commercialization; Drug development; Drug screening; Fractures; Fragile X syndrome; FRAX; Inhibitory postsynaptic potentials; Intellectual disabilities; iPSCs; Mutation; Neurological diseases; Neurological disorders; Parkinson's disease; Pluripotency; Review; Risk assessment; Stem cell transplantation; Stem cells; ALS; drug development; iPSCs; FRAX; AD; PD
• ISSN: 1422-0067; 1661-6596; 1422-0067
• DOI: 10.3390/ijms22094596

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).