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Literature DB >> 22146428

Diseases in a dish: modeling human genetic disorders using induced pluripotent cells.

Gustavo Tiscornia¹, Erica Lorenzo Vivas, Juan Carlos Izpisúa Belmonte.

Abstract

The derivation of induced pluripotent cells (iPSCs) from individuals suffering from genetic syndromes offers new opportunities for basic research into these diseases and the development of therapeutic compounds. iPSCs can self renew and can be differentiated to many cell types, offering a potentially unlimited source of material for study. In this review we discuss the conceptual and practical issues to consider when attempting to model genetic diseases using iPSCs.

Entities: Disease Species

Mesh：

Year: 2011 PMID： 22146428 DOI： 10.1038/nm.2504

Source DB: PubMed Journal: Nat Med ISSN： 1078-8956 Impact factor: 53.440

38 in total

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Journal: BMJ Date: 2006-12-15

3. 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

Review 4. A critical appraisal of the NXY-059 neuroprotection studies for acute stroke: a need for more rigorous testing of neuroprotective agents in animal models of stroke.

Authors: Sean I Savitz
Journal: Exp Neurol Date: 2007-03-12 Impact factor: 5.330

5. Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs.

Authors: Guang-Hui Liu; Keiichiro Suzuki; Jing Qu; Ignacio Sancho-Martinez; Fei Yi; Mo Li; Sachin Kumar; Emmanuel Nivet; Jessica Kim; Rupa Devi Soligalla; Ilir Dubova; April Goebl; Nongluk Plongthongkum; Ho-Lim Fung; Kun Zhang; Jeanne F Loring; Louise C Laurent; Juan Carlos Izpisua Belmonte
Journal: Cell Stem Cell Date: 2011-05-19 Impact factor: 24.633

Review 6. Technical challenges in using human induced pluripotent stem cells to model disease.

Authors: Krishanu Saha; Rudolf Jaenisch
Journal: Cell Stem Cell Date: 2009-12-04 Impact factor: 24.633

7. Induced pluripotent stem cells from a spinal muscular atrophy patient.

Authors: Allison D Ebert; Junying Yu; Ferrill F Rose; Virginia B Mattis; Christian L Lorson; James A Thomson; Clive N Svendsen
Journal: Nature Date: 2008-12-21 Impact factor: 49.962

8. Embryonic stem cell lines derived from human blastocysts.

Authors: J A Thomson; J Itskovitz-Eldor; S S Shapiro; M A Waknitz; J J Swiergiel; V S Marshall; J M Jones
Journal: Science Date: 1998-11-06 Impact factor: 47.728

9. Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells.

Authors: Angel Raya; Ignasi Rodríguez-Pizà; Guillermo Guenechea; Rita Vassena; Susana Navarro; María José Barrero; Antonella Consiglio; Maria Castellà; Paula Río; Eduard Sleep; Federico González; Gustavo Tiscornia; Elena Garreta; Trond Aasen; Anna Veiga; Inder M Verma; Jordi Surrallés; Juan Bueren; Juan Carlos Izpisúa Belmonte
Journal: Nature Date: 2009-05-31 Impact factor: 49.962

10. Modelling pathogenesis and treatment of familial dysautonomia using patient-specific iPSCs.

Authors: Gabsang Lee; Eirini P Papapetrou; Hyesoo Kim; Stuart M Chambers; Mark J Tomishima; Christopher A Fasano; Yosif M Ganat; Jayanthi Menon; Fumiko Shimizu; Agnes Viale; Viviane Tabar; Michel Sadelain; Lorenz Studer
Journal: Nature Date: 2009-08-19 Impact factor: 49.962

84 in total

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Review 2. In vitro models of the blood-brain barrier: An overview of commonly used brain endothelial cell culture models and guidelines for their use.

Authors: Hans C Helms; N Joan Abbott; Malgorzata Burek; Romeo Cecchelli; Pierre-Olivier Couraud; Maria A Deli; Carola Förster; Hans J Galla; Ignacio A Romero; Eric V Shusta; Matthew J Stebbins; Elodie Vandenhaute; Babette Weksler; Birger Brodin
Journal: J Cereb Blood Flow Metab Date: 2016-02-11 Impact factor: 6.200

Review 3. Genome-wide association studies of late-onset cardiovascular disease.

Authors: J Gustav Smith; Christopher Newton-Cheh
Journal: J Mol Cell Cardiol Date: 2015-04-11 Impact factor: 5.000

Review 4. Progress and prospects for genetic modification of nonhuman primate models in biomedical research.

Authors: Anthony W S Chan
Journal: ILAR J Date: 2013

5. Derivation of induced pluripotent stem cells from ferret somatic cells.

Authors: Jinghui Gao; Sophia Petraki; Xingshen Sun; Leonard A Brooks; Thomas J Lynch; Chih-Lin Hsieh; Reem Elteriefi; Zareeb Lorenzana; Vasu Punj; John F Engelhardt; Kalpaj R Parekh; Amy L Ryan
Journal: Am J Physiol Lung Cell Mol Physiol Date: 2020-02-19 Impact factor: 5.464

10. Neural progenitors derived from Tuberous Sclerosis Complex patients exhibit attenuated PI3K/AKT signaling and delayed neuronal differentiation.

Authors: Avery J Zucco; Valentina Dal Pozzo; Alina Afinogenova; Ronald P Hart; Orrin Devinsky; Gabriella D'Arcangelo
Journal: Mol Cell Neurosci Date: 2018-08-23 Impact factor: 4.314