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Literature DB >> 26514199

Clinical Trials in a Dish: The Potential of Pluripotent Stem Cells to Develop Therapies for Neurodegenerative Diseases.

Kelly M Haston¹, Steven Finkbeiner^1,2,3.

Abstract

Neurodegenerative diseases are a leading cause of death. No disease-modifying therapies are available, and preclinical animal model data have routinely failed to translate into success for therapeutics. Induced pluripotent stem cell (iPSC) biology holds great promise for human in vitro disease modeling because these cells can give rise to any cell in the human brain and display phenotypes specific to neurodegenerative diseases previously identified in postmortem and clinical samples. Here, we explore the potential and caveats of iPSC technology as a platform for drug development and screening, and the future potential to use large cohorts of disease-bearing iPSCs to perform clinical trials in a dish.

Entities: CellLine Chemical Disease Gene Species

Keywords: clinical trials; drug development; human pluripotent stem cells; neurodegeneration

Mesh：

Year: 2015 PMID： 26514199 PMCID： PMC4868344 DOI： 10.1146/annurev-pharmtox-010715-103548

Source DB: PubMed Journal: Annu Rev Pharmacol Toxicol ISSN： 0362-1642 Impact factor: 13.820

146 in total

1. Intermediate filament protein accumulation in motor neurons derived from giant axonal neuropathy iPSCs rescued by restoration of gigaxonin.

Authors: Bethany L Johnson-Kerner; Faizzan S Ahmad; Alejandro Garcia Diaz; John Palmer Greene; Steven J Gray; Richard Jude Samulski; Wendy K Chung; Rudy Van Coster; Paul Maertens; Scott A Noggle; Christopher E Henderson; Hynek Wichterle
Journal: Hum Mol Genet Date: 2014-11-04 Impact factor: 6.150

Review 2. Genetic modifiers of Huntington's disease.

Authors: James F Gusella; Marcy E MacDonald; Jong-Min Lee
Journal: Mov Disord Date: 2014-08-25 Impact factor: 10.338

3. Loss of spastin function results in disease-specific axonal defects in human pluripotent stem cell-based models of hereditary spastic paraplegia.

Authors: Kyle R Denton; Ling Lei; Jeremy Grenier; Vladimir Rodionov; Craig Blackstone; Xue-Jun Li
Journal: Stem Cells Date: 2014-02 Impact factor: 6.277

4. Adapting human pluripotent stem cells to high-throughput and high-content screening.

Authors: Sabrina C Desbordes; Lorenz Studer
Journal: Nat Protoc Date: 2012-12-20 Impact factor: 13.491

5. Cytoplasmic mislocalization of TDP-43 is toxic to neurons and enhanced by a mutation associated with familial amyotrophic lateral sclerosis.

Authors: Sami J Barmada; Gaia Skibinski; Erica Korb; Elizabeth J Rao; Jane Y Wu; Steven Finkbeiner
Journal: J Neurosci Date: 2010-01-13 Impact factor: 6.167

6. Modeling ALS with iPSCs reveals that mutant SOD1 misregulates neurofilament balance in motor neurons.

Authors: Hong Chen; Kun Qian; Zhongwei Du; Jingyuan Cao; Andrew Petersen; Huisheng Liu; Lisle W Blackbourn; CindyTzu-Ling Huang; Anthony Errigo; Yingnan Yin; Jianfeng Lu; Melvin Ayala; Su-Chun Zhang
Journal: Cell Stem Cell Date: 2014-04-03 Impact factor: 24.633

7. Somatic coding mutations in human induced pluripotent stem cells.

Authors: Athurva Gore; Zhe Li; Ho-Lim Fung; Jessica E Young; Suneet Agarwal; Jessica Antosiewicz-Bourget; Isabel Canto; Alessandra Giorgetti; Mason A Israel; Evangelos Kiskinis; Je-Hyuk Lee; Yuin-Han Loh; Philip D Manos; Nuria Montserrat; Athanasia D Panopoulos; Sergio Ruiz; Melissa L Wilbert; Junying Yu; Ewen F Kirkness; Juan Carlos Izpisua Belmonte; Derrick J Rossi; James A Thomson; Kevin Eggan; George Q Daley; Lawrence S B Goldstein; Kun Zhang
Journal: Nature Date: 2011-03-03 Impact factor: 49.962

8. Developmentally coordinated extrinsic signals drive human pluripotent stem cell differentiation toward authentic DARPP-32+ medium-sized spiny neurons.

Authors: Alessia Delli Carri; Marco Onorati; Mariah J Lelos; Valentina Castiglioni; Andrea Faedo; Ramesh Menon; Stefano Camnasio; Romina Vuono; Paolo Spaiardi; Francesca Talpo; Mauro Toselli; Gianvito Martino; Roger A Barker; Stephen B Dunnett; Gerardo Biella; Elena Cattaneo
Journal: Development Date: 2013-01-15 Impact factor: 6.868

9. Autophagy induction enhances TDP43 turnover and survival in neuronal ALS models.

Authors: Sami J Barmada; Andrea Serio; Arpana Arjun; Bilada Bilican; Aaron Daub; D Michael Ando; Andrey Tsvetkov; Michael Pleiss; Xingli Li; Daniel Peisach; Christopher Shaw; Siddharthan Chandran; Steven Finkbeiner
Journal: Nat Chem Biol Date: 2014-06-29 Impact factor: 15.040

Review 10. Huntington's disease: the past, present, and future search for disease modifiers.

Authors: Erin B D Clabough
Journal: Yale J Biol Med Date: 2013-06-13

24 in total

1. Three-Dimensional Hyaluronic Acid Hydrogel-Based Models for In Vitro Human iPSC-Derived NPC Culture and Differentiation.

Authors: Shaohua Wu; Ranjie Xu; Bin Duan; Peng Jiang
Journal: J Mater Chem B Date: 2017-04-19 Impact factor: 6.331

Review 2. Cell death assays for neurodegenerative disease drug discovery.

Authors: Jeremy W Linsley; Terry Reisine; Steven Finkbeiner
Journal: Expert Opin Drug Discov Date: 2019-06-10 Impact factor: 6.098

Review 3. Genetic predispositions of Parkinson's disease revealed in patient-derived brain cells.

Authors: Jenne Tran; Helena Anastacio; Cedric Bardy
Journal: NPJ Parkinsons Dis Date: 2020-04-24

Review 4. iPS cells in the study of PD molecular pathogenesis.

Authors: Melanie M Cobb; Abinaya Ravisankar; Gaia Skibinski; Steven Finkbeiner
Journal: Cell Tissue Res Date: 2017-12-12 Impact factor: 5.249

5. Small-scale Propagation of Human iPSCs in Serum-free Conditions for Routine Immunocytochemical Characterization.

Authors: Mandi J Corenblum; Lalitha Madhavan
Journal: J Vis Exp Date: 2017-02-18 Impact factor: 1.355

6. CD49f Is a Novel Marker of Functional and Reactive Human iPSC-Derived Astrocytes.

Authors: Lilianne Barbar; Tanya Jain; Matthew Zimmer; Ilya Kruglikov; Jessica S Sadick; Minghui Wang; Kriti Kalpana; Indigo V L Rose; Suzanne R Burstein; Tomasz Rusielewicz; Madhura Nijsure; Kevin A Guttenplan; Angelique di Domenico; Gist Croft; Bin Zhang; Hiroko Nobuta; Jean M Hébert; Shane A Liddelow; Valentina Fossati
Journal: Neuron Date: 2020-06-01 Impact factor: 17.173