Literature DB >> 29771306

Drug screening for human genetic diseases using iPSC models.

Matthew S Elitt1, Lilianne Barbar1, Paul J Tesar1.   

Abstract

Induced pluripotent stem cells (iPSCs) enable the generation of previously unattainable, scalable quantities of disease-relevant tissues from patients suffering from essentially any genetic disorder. This cellular material has proven instrumental for drug screening efforts on these disorders, and has facilitated the identification of novel therapeutics for patients. Here we will review the foundational technologies that have enabled iPSCs, the power and limitations of iPSC-based compound screens along with screening guidelines, and recent examples of screening efforts. Additionally we will provide a brief commentary on the future scientific roadmap using pluripotent- and 3D organoid-based, combinatorial approaches.

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Mesh：

Year:  2018        PMID： 29771306      PMCID： PMC6061782          DOI： 10.1093/hmg/ddy186

Source DB:  PubMed          Journal:  Hum Mol Genet        ISSN： 0964-6906            Impact factor:   6.150


  130 in total

1.  Directing human embryonic stem cell differentiation towards a renal lineage generates a self-organizing kidney.

Authors:  M Takasato; P X Er; M Becroft; J M Vanslambrouck; E G Stanley; A G Elefanty; M H Little
Journal:  Nat Cell Biol       Date:  2013-12-15       Impact factor: 28.824

2.  Viable offspring derived from fetal and adult mammalian cells.

Authors:  I Wilmut; A E Schnieke; J McWhir; A J Kind; K H Campbell
Journal:  Nature       Date:  1997-02-27       Impact factor: 49.962

Review 3.  Disease Modeling in Stem Cell-Derived 3D Organoid Systems.

Authors:  Devanjali Dutta; Inha Heo; Hans Clevers
Journal:  Trends Mol Med       Date:  2017-03-21       Impact factor: 11.951

4.  Human Astrocyte Maturation Captured in 3D Cerebral Cortical Spheroids Derived from Pluripotent Stem Cells.

Authors:  Steven A Sloan; Spyros Darmanis; Nina Huber; Themasap A Khan; Fikri Birey; Christine Caneda; Richard Reimer; Stephen R Quake; Ben A Barres; Sergiu P Paşca
Journal:  Neuron       Date:  2017-08-16       Impact factor: 17.173

5.  An Organoid-Based Model of Cortical Development Identifies Non-Cell-Autonomous Defects in Wnt Signaling Contributing to Miller-Dieker Syndrome.

Authors:  Vira Iefremova; George Manikakis; Olivia Krefft; Ammar Jabali; Kevin Weynans; Ruven Wilkens; Fabio Marsoner; Björn Brändl; Franz-Josef Müller; Philipp Koch; Julia Ladewig
Journal:  Cell Rep       Date:  2017-04-04       Impact factor: 9.423

Review 6.  Engineering Stem Cell Organoids.

Authors:  Xiaolei Yin; Benjamin E Mead; Helia Safaee; Robert Langer; Jeffrey M Karp; Oren Levy
Journal:  Cell Stem Cell       Date:  2016-01-07       Impact factor: 24.633

7.  Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9.

Authors:  Dominik Paquet; Dylan Kwart; Antonia Chen; Andrew Sproul; Samson Jacob; Shaun Teo; Kimberly Moore Olsen; Andrew Gregg; Scott Noggle; Marc Tessier-Lavigne
Journal:  Nature       Date:  2016-04-27       Impact factor: 49.962

Review 8.  Genetic and epigenetic variations in iPSCs: potential causes and implications for application.

Authors:  Gaoyang Liang; Yi Zhang
Journal:  Cell Stem Cell       Date:  2013-08-01       Impact factor: 24.633

9.  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

10.  Direct cell reprogramming is a stochastic process amenable to acceleration.

Authors:  Jacob Hanna; Krishanu Saha; Bernardo Pando; Jeroen van Zon; Christopher J Lengner; Menno P Creyghton; Alexander van Oudenaarden; Rudolf Jaenisch
Journal:  Nature       Date:  2009-11-08       Impact factor: 49.962
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  42 in total

1.  Production and cryopreservation of definitive endoderm from human pluripotent stem cells under defined and scalable culture conditions.

Authors:  Anais Sahabian; Julia Dahlmann; Ulrich Martin; Ruth Olmer
Journal:  Nat Protoc       Date:  2021-02-12       Impact factor: 13.491

2.  Challenges and Opportunities for Translation of Therapies to Improve Cognition in Down Syndrome.

Authors:  Sarah E Lee; Monica Duran-Martinez; Sabina Khantsis; Diana W Bianchi; Faycal Guedj
Journal:  Trends Mol Med       Date:  2019-11-07       Impact factor: 11.951

3.  Directing differentiation of human induced pluripotent stem cells toward androgen-producing Leydig cells rather than adrenal cells.

Authors:  Lu Li; Yuchang Li; Chantal Sottas; Martine Culty; Jinjiang Fan; Yiman Hu; Garett Cheung; Héctor E Chemes; Vassilios Papadopoulos
Journal:  Proc Natl Acad Sci U S A       Date:  2019-10-07       Impact factor: 11.205

4.  Generation of Human iPSC from Small Volume Peripheral Blood Samples.

Authors:  Doreen Kloos; Nico Lachmann
Journal:  Methods Mol Biol       Date:  2022

5.  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

Review 6.  Therapies for rare diseases: therapeutic modalities, progress and challenges ahead.

Authors:  Erik Tambuyzer; Benjamin Vandendriessche; Christopher P Austin; Philip J Brooks; Kristina Larsson; Katherine I Miller Needleman; James Valentine; Kay Davies; Stephen C Groft; Robert Preti; Tudor I Oprea; Marco Prunotto
Journal:  Nat Rev Drug Discov       Date:  2019-12-13       Impact factor: 84.694

7.  A low-cost 3D printed microfluidic bioreactor and imaging chamber for live-organoid imaging.

Authors:  Ikram Khan; Anil Prabhakar; Chloe Delepine; Hayley Tsang; Vincent Pham; Mriganka Sur
Journal:  Biomicrofluidics       Date:  2021-04-06       Impact factor: 2.800

8.  iPSC-endothelial cell phenotypic drug screening and in silico analyses identify tyrphostin-AG1296 for pulmonary arterial hypertension.

Authors:  Mingxia Gu; Michele Donato; Minzhe Guo; Neil Wary; Yifei Miao; Shuai Mao; Toshie Saito; Shoichiro Otsuki; Lingli Wang; Rebecca L Harper; Silin Sa; Purvesh Khatri; Marlene Rabinovitch
Journal:  Sci Transl Med       Date:  2021-05-05       Impact factor: 17.956

Review 9.  The Path to Progress Preclinical Studies of Age-Related Neurodegenerative Diseases: A Perspective on Rodent and hiPSC-Derived Models.

Authors:  Gabriella MacDougall; Logan Y Brown; Boris Kantor; Ornit Chiba-Falek
Journal:  Mol Ther       Date:  2021-01-09       Impact factor: 11.454

Review 10.  Advances in stem cell research for the treatment of primary hypogonadism.

Authors:  Lu Li; Vassilios Papadopoulos
Journal:  Nat Rev Urol       Date:  2021-06-29       Impact factor: 14.432
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