Literature DB >> 28478951

A CRISPR Approach to Neurodegenerative Diseases.

Martin Kampmann1.   

Abstract

A major barrier to developing effective therapies for neurodegenerative diseases is our incomplete understanding of the underlying cellular mechanisms. Genetic screens in human-induced pluripotent stem cell-derived neurons can elucidate such mechanisms. Genome-wide screens using CRISPR interference and CRISPR activation provide complementary biological insights and may reveal potential therapeutic targets.
Copyright © 2017 Elsevier Ltd. All rights reserved.

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Year:  2017        PMID: 28478951      PMCID: PMC5604856          DOI: 10.1016/j.molmed.2017.04.003

Source DB:  PubMed          Journal:  Trends Mol Med        ISSN: 1471-4914            Impact factor:   11.951


  11 in total

1.  LRRK2 mutant iPSC-derived DA neurons demonstrate increased susceptibility to oxidative stress.

Authors:  Ha Nam Nguyen; Blake Byers; Branden Cord; Aleksandr Shcheglovitov; James Byrne; Prachi Gujar; Kehkooi Kee; Birgitt Schüle; Ricardo E Dolmetsch; William Langston; Theo D Palmer; Renee Reijo Pera
Journal:  Cell Stem Cell       Date:  2011-03-04       Impact factor: 24.633

2.  Next-generation libraries for robust RNA interference-based genome-wide screens.

Authors:  Martin Kampmann; Max A Horlbeck; Yuwen Chen; Jordan C Tsai; Michael C Bassik; Luke A Gilbert; Jacqueline E Villalta; S Chul Kwon; Hyeshik Chang; V Narry Kim; Jonathan S Weissman
Journal:  Proc Natl Acad Sci U S A       Date:  2015-06-15       Impact factor: 11.205

3.  CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs.

Authors:  Mohammad A Mandegar; Nathaniel Huebsch; Ekaterina B Frolov; Edward Shin; Annie Truong; Michael P Olvera; Amanda H Chan; Yuichiro Miyaoka; Kristin Holmes; C Ian Spencer; Luke M Judge; David E Gordon; Tilde V Eskildsen; Jacqueline E Villalta; Max A Horlbeck; Luke A Gilbert; Nevan J Krogan; Søren P Sheikh; Jonathan S Weissman; Lei S Qi; Po-Lin So; Bruce R Conklin
Journal:  Cell Stem Cell       Date:  2016-03-10       Impact factor: 24.633

4.  Integrated platform for genome-wide screening and construction of high-density genetic interaction maps in mammalian cells.

Authors:  Martin Kampmann; Michael C Bassik; Jonathan S Weissman
Journal:  Proc Natl Acad Sci U S A       Date:  2013-06-05       Impact factor: 11.205

5.  Functional genomics of brain aging and Alzheimer's disease: focus on selective neuronal vulnerability.

Authors:  Xinkun Wang; Mary L Michaelis; Elias K Michaelis
Journal:  Curr Genomics       Date:  2010-12       Impact factor: 2.236

6.  Pharmacological dimerization and activation of the exchange factor eIF2B antagonizes the integrated stress response.

Authors:  Carmela Sidrauski; Jordan C Tsai; Martin Kampmann; Brian R Hearn; Punitha Vedantham; Priyadarshini Jaishankar; Masaaki Sokabe; Aaron S Mendez; Billy W Newton; Edward L Tang; Erik Verschueren; Jeffrey R Johnson; Nevan J Krogan; Christopher S Fraser; Jonathan S Weissman; Adam R Renslo; Peter Walter
Journal:  Elife       Date:  2015-04-15       Impact factor: 8.140

7.  Molecular biology. Use and abuse of RNAi to study mammalian gene function.

Authors:  William G Kaelin
Journal:  Science       Date:  2012-07-27       Impact factor: 47.728

8.  Probing sporadic and familial Alzheimer's disease using induced pluripotent stem cells.

Authors:  Mason A Israel; Shauna H Yuan; Cedric Bardy; Sol M Reyna; Yangling Mu; Cheryl Herrera; Michael P Hefferan; Sebastiaan Van Gorp; Kristopher L Nazor; Francesca S Boscolo; Christian T Carson; Louise C Laurent; Martin Marsala; Fred H Gage; Anne M Remes; Edward H Koo; Lawrence S B Goldstein
Journal:  Nature       Date:  2012-01-25       Impact factor: 49.962

9.  Compact and highly active next-generation libraries for CRISPR-mediated gene repression and activation.

Authors:  Max A Horlbeck; Luke A Gilbert; Jacqueline E Villalta; Britt Adamson; Ryan A Pak; Yuwen Chen; Alexander P Fields; Chong Yon Park; Jacob E Corn; Martin Kampmann; Jonathan S Weissman
Journal:  Elife       Date:  2016-09-23       Impact factor: 8.140

10.  Genome-Scale CRISPR-Mediated Control of Gene Repression and Activation.

Authors:  Luke A Gilbert; Max A Horlbeck; Britt Adamson; Jacqueline E Villalta; Yuwen Chen; Evan H Whitehead; Carla Guimaraes; Barbara Panning; Hidde L Ploegh; Michael C Bassik; Lei S Qi; Martin Kampmann; Jonathan S Weissman
Journal:  Cell       Date:  2014-10-09       Impact factor: 41.582

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  15 in total

1.  CRISPR Interference-Based Platform for Multimodal Genetic Screens in Human iPSC-Derived Neurons.

Authors:  Ruilin Tian; Mariam A Gachechiladze; Connor H Ludwig; Matthew T Laurie; Jason Y Hong; Diane Nathaniel; Anika V Prabhu; Michael S Fernandopulle; Rajan Patel; Mehrnoosh Abshari; Michael E Ward; Martin Kampmann
Journal:  Neuron       Date:  2019-08-15       Impact factor: 17.173

2.  Compromised function of the ESCRT pathway promotes endolysosomal escape of tau seeds and propagation of tau aggregation.

Authors:  John J Chen; Diane L Nathaniel; Preethi Raghavan; Maxine Nelson; Ruilin Tian; Eric Tse; Jason Y Hong; Stephanie K See; Sue-Ann Mok; Marco Y Hein; Daniel R Southworth; Lea T Grinberg; Jason E Gestwicki; Manuel D Leonetti; Martin Kampmann
Journal:  J Biol Chem       Date:  2019-10-02       Impact factor: 5.157

Review 3.  CRISPRi and CRISPRa Screens in Mammalian Cells for Precision Biology and Medicine.

Authors:  Martin Kampmann
Journal:  ACS Chem Biol       Date:  2017-10-24       Impact factor: 5.100

Review 4.  Emerging Therapies and Novel Targets for TDP-43 Proteinopathy in ALS/FTD.

Authors:  Lindsey R Hayes; Petr Kalab
Journal:  Neurotherapeutics       Date:  2022-07-05       Impact factor: 6.088

Review 5.  Exploring the Involvement of the Amyloid Precursor Protein A673T Mutation against Amyloid Pathology and Alzheimer's Disease in Relation to Therapeutic Editing Tools.

Authors:  Gabriela Dumitrita Stanciu; Daniela Carmen Ababei; Razvan Nicolae Rusu; Veronica Bild; Bogdan-Ionel Tamba
Journal:  Pharmaceutics       Date:  2022-06-15       Impact factor: 6.525

6.  CRISPulator: a discrete simulation tool for pooled genetic screens.

Authors:  Tamas Nagy; Martin Kampmann
Journal:  BMC Bioinformatics       Date:  2017-07-21       Impact factor: 3.169

Review 7.  Editing the Central Nervous System Through CRISPR/Cas9 Systems.

Authors:  Agustin Cota-Coronado; Néstor Fabián Díaz-Martínez; Eduardo Padilla-Camberos; N Emmanuel Díaz-Martínez
Journal:  Front Mol Neurosci       Date:  2019-05-27       Impact factor: 5.639

Review 8.  CRISPR-based functional genomics for neurological disease.

Authors:  Martin Kampmann
Journal:  Nat Rev Neurol       Date:  2020-07-08       Impact factor: 42.937

Review 9.  Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation.

Authors:  Aino Vesikansa
Journal:  J Cent Nerv Syst Dis       Date:  2018-07-10

Review 10.  Methods and applications of CRISPR/Cas system for genome editing in stem cells.

Authors:  Guang Yang; Xingxu Huang
Journal:  Cell Regen       Date:  2019-10-11
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