Literature DB >> 34595731

Screening Platforms for Genetic Epilepsies-Zebrafish, iPSC-Derived Neurons, and Organoids.

Aleksandr Shcheglovitov1, Randall T Peterson2.   

Abstract

Recent advances in molecular and cellular engineering, such as human cell reprogramming, genome editing, and patient-specific organoids, have provided unprecedented opportunities for investigating human disorders in both animals and human-based models at an improved pace and precision. This progress will inevitably lead to the development of innovative drug-screening platforms and new patient-specific therapeutics. In this review, we discuss recent advances that have been made using zebrafish and human-induced pluripotent stem cell (iPSC)-derived neurons and organoids for modeling genetic epilepsies. We also provide our prospective on how these models can potentially be combined to build new screening platforms for antiseizure and antiepileptogenic drug discovery that harness the robustness and tractability of zebrafish models as well as the patient-specific genetics and biology of iPSC-derived neurons and organoids.
© 2021. The American Society for Experimental NeuroTherapeutics, Inc.

Entities:  

Keywords:  Developmental and epileptic encephalopathy (DEE); Drug discovery; Epilepsy; Organoids; Stem cells; Zebrafish; iPSC

Mesh:

Substances:

Year:  2021        PMID: 34595731      PMCID: PMC8608971          DOI: 10.1007/s13311-021-01115-5

Source DB:  PubMed          Journal:  Neurotherapeutics        ISSN: 1878-7479            Impact factor:   6.088


  131 in total

1.  MicroRNA-mediated conversion of human fibroblasts to neurons.

Authors:  Andrew S Yoo; Alfred X Sun; Li Li; Aleksandr Shcheglovitov; Thomas Portmann; Yulong Li; Chris Lee-Messer; Ricardo E Dolmetsch; Richard W Tsien; Gerald R Crabtree
Journal:  Nature       Date:  2011-07-13       Impact factor: 49.962

Review 2.  Generation of cerebral cortical GABAergic interneurons from pluripotent stem cells.

Authors:  Megan Fitzgerald; Nathaniel Sotuyo; David J Tischfield; Stewart A Anderson
Journal:  Stem Cells       Date:  2020-09-02       Impact factor: 6.277

3.  Complex Oscillatory Waves Emerging from Cortical Organoids Model Early Human Brain Network Development.

Authors:  Cleber A Trujillo; Richard Gao; Priscilla D Negraes; Jing Gu; Justin Buchanan; Sebastian Preissl; Allen Wang; Wei Wu; Gabriel G Haddad; Isaac A Chaim; Alain Domissy; Matthieu Vandenberghe; Anna Devor; Gene W Yeo; Bradley Voytek; Alysson R Muotri
Journal:  Cell Stem Cell       Date:  2019-08-29       Impact factor: 24.633

4.  Ultra-Rare Genetic Variation in the Epilepsies: A Whole-Exome Sequencing Study of 17,606 Individuals.

Authors: 
Journal:  Am J Hum Genet       Date:  2019-07-18       Impact factor: 11.025

Review 5.  Probing disrupted neurodevelopment in autism using human stem cell-derived neurons and organoids: An outlook into future diagnostics and drug development.

Authors:  Guang Yang; Alex Shcheglovitov
Journal:  Dev Dyn       Date:  2019-10-01       Impact factor: 3.780

Review 6.  Therapeutic advances in Dravet syndrome: a targeted literature review.

Authors:  Adam Strzelczyk; Susanne Schubert-Bast
Journal:  Expert Rev Neurother       Date:  2020-08-16       Impact factor: 4.618

7.  De novo gain-of-function KCNT1 channel mutations cause malignant migrating partial seizures of infancy.

Authors:  Giulia Barcia; Matthew R Fleming; Aline Deligniere; Valeswara-Rao Gazula; Maile R Brown; Maeva Langouet; Haijun Chen; Jack Kronengold; Avinash Abhyankar; Roberta Cilio; Patrick Nitschke; Anna Kaminska; Nathalie Boddaert; Jean-Laurent Casanova; Isabelle Desguerre; Arnold Munnich; Olivier Dulac; Leonard K Kaczmarek; Laurence Colleaux; Rima Nabbout
Journal:  Nat Genet       Date:  2012-10-21       Impact factor: 38.330

8.  Functional and Mechanistic Neurotoxicity Profiling Using Human iPSC-Derived Neural 3D Cultures.

Authors:  Oksana Sirenko; Frederick Parham; Steven Dea; Neha Sodhi; Steven Biesmans; Sergio Mora-Castilla; Kristen Ryan; Mamta Behl; Grischa Chandy; Carole Crittenden; Sarah Vargas-Hurlston; Oivin Guicherit; Ryan Gordon; Fabian Zanella; Cassiano Carromeu
Journal:  Toxicol Sci       Date:  2019-01-01       Impact factor: 4.849

9.  Dravet Syndrome: A Developmental and Epileptic Encephalopathy.

Authors:  Luis Lopez-Santiago; Lori L Isom
Journal:  Epilepsy Curr       Date:  2019-01-30       Impact factor: 7.500

10.  Efficient genome editing in zebrafish using a CRISPR-Cas system.

Authors:  Woong Y Hwang; Yanfang Fu; Deepak Reyon; Morgan L Maeder; Shengdar Q Tsai; Jeffry D Sander; Randall T Peterson; J-R Joanna Yeh; J Keith Joung
Journal:  Nat Biotechnol       Date:  2013-01-29       Impact factor: 54.908

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  5 in total

Review 1.  Neurodevelopmental copy-number variants: A roadmap to improving outcomes by uniting patient advocates, researchers, and clinicians for collective impact.

Authors: 
Journal:  Am J Hum Genet       Date:  2022-08-04       Impact factor: 11.043

2.  Precision Treatments in Epilepsy.

Authors:  Scott Demarest; Amy Brooks-Kayal
Journal:  Neurotherapeutics       Date:  2021-10-26       Impact factor: 6.088

3.  Epilepsy Research Now in 3D: Harnessing the Power of Brain Organoids in Epilepsy.

Authors:  Christina Gross
Journal:  Epilepsy Curr       Date:  2022-01-06       Impact factor: 7.500

Review 4.  Functional Characterization of Human Pluripotent Stem Cell-Derived Models of the Brain with Microelectrode Arrays.

Authors:  Anssi Pelkonen; Cristiana Pistono; Pamela Klecki; Mireia Gómez-Budia; Antonios Dougalis; Henna Konttinen; Iveta Stanová; Ilkka Fagerlund; Ville Leinonen; Paula Korhonen; Tarja Malm
Journal:  Cells       Date:  2021-12-29       Impact factor: 6.600

Review 5.  Teleost Fish and Organoids: Alternative Windows Into the Development of Healthy and Diseased Brains.

Authors:  Giulia Fasano; Claudia Compagnucci; Bruno Dallapiccola; Marco Tartaglia; Antonella Lauri
Journal:  Front Mol Neurosci       Date:  2022-08-11       Impact factor: 6.261

  5 in total

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