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Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders

Polit, Lucia Dutan, Eidhof, Ilse, McNeill, Rhiannon V., Warre-Cornish, Katherine M., Ohki, Cristine Marie Yde, Walter, Natalie Monet, Sala, Carlo, Verpelli, Chiara, Radtke, Franziska, Galderisi, Silvana, Mucci, Armida, Collo, Ginetta, Edenhofer, Frank, Castrén, Maija L., Réthelyi, János M., Ejlersen, Morten, Hohmann, Sonja Simone, Ilieva, Mirolyuba S., Lukjanska, Renate, Matuleviciute, Rugile, Michel, Tanja Maria, de Vrij, Femke M.S., Kushner, Steven A., Lendemeijer, Bas, Kittel-Schneider, Sarah, Ziegler, Georg C., Gruber-Schoffnegger, Doris, Pasterkamp, R. Jeroen, Kasri, Amal, Potier, Marie-Claude, Knoblich, Jürgen A., Brüstle, Oliver, Peitz, Michael, Pich, Emilio Merlo, Harwood, Adrian J. ORCID:, Abranches, Elsa, Falk, Anna, Vernon, Anthony C., Grünblatt, Edna and Srivastava, Deepak P. 2023. Recommendations, guidelines, and best practice for the use of human induced pluripotent stem cells for neuropharmacological studies of neuropsychiatric disorders. Neuroscience Applied 2 , 101125. 10.1016/j.nsa.2023.101125

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The number of individuals suffering from neuropsychiatric disorders (NPDs) has increased worldwide, with 3 million disability-adjusted life-years calculated in 2019. Though research using various approaches including genetics, imaging, clinical and animal models has advanced our knowledge regarding NPDs, we still lack basic knowledge regarding the underlying pathophysiological mechanisms. Moreover, there is an urgent need for highly effective therapeutics for NPDs i. Human induced pluripotent stem cells (hiPSCs) generated from somatic cells enabled scientists to create brain cells in a patient-specific manner. However, there are challenges to the use of hiPSCs that need to be addressed. In the current paper, consideration of best practices for neuropharmacological and neuropsychiatric research using hiPSCs will be discussed. Specifically, we provide recommendations for best practice in patient recruitment, including collecting demographic, clinical, medical (before and after treatment and response), diagnostic (incl. scales) and genetic data from the donors. We highlight considerations regarding donor genetics and sex, in addition to discussing biological and technical replicates. Furthermore, we present our views on selecting control groups/lines, experimental designs, and considerations for conducting neuropharmacological studies using hiPSC-based models in the context of NPDs. In doing so, we explore key issues in the field concerning reproducibility, statistical analysis, and how to translate in vitro studies into clinically relevant observations. The aim of this article is to provide a key resource for hiPSC researchers to perform robust and reproducible neuropharmacological studies, with the ultimate aim of improving identification and clinical translation of novel therapeutic drugs for NPDs.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Biosciences
Neuroscience and Mental Health Research Institute (NMHRI)
Additional Information: License information from Publisher: LICENSE 1: URL:, Start Date: 2023-03-16
Publisher: Elsevier
ISSN: 2772-4085
Date of First Compliant Deposit: 23 March 2023
Date of Acceptance: 15 March 2023
Last Modified: 27 Feb 2024 12:28

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