ArticleNucleic acids research2015
Genome engineering of isogenic human ES cells to model autism disorders.
Article in Nucleic acids research, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
12 citing papers in PubMed, 17 citations in OpenAlex.
- hPSC-Derived Astrocytes at the Forefront of Translational Applications in Neurological Disorders.Cells · 2024Review
- CRISPR/Cas-Based Approaches to Study Schizophrenia and Other Neurodevelopmental Disorders.International journal of molecular sciences · 2022Review
- Modeling Psychiatric Disorder Biology with Stem Cells.Current psychiatry reports · 2020Review
- Dysfunctional Cav1.2 channel in Timothy syndrome, from cell to bedside.Experimental biology and medicine (Maywood, N.J.) · 2019Article
- Generation of an oxoglutarate dehydrogenase knockout rat model and the effect of a high-fat diet.RSC advances · 2018Article
- Genome editing: a robust technology for human stem cells.Cellular and molecular life sciences : CMLS · 2017Review
- A Single-Cell Roadmap of Lineage Bifurcation in Human ESC Models of Embryonic Brain Development.Cell stem cell · 2017Article
- To CRISPR and beyond: the evolution of genome editing in stem cells.Regenerative medicine · 2016Review
- Spatial genome organization and cognition.Nature reviews. Neuroscience · 2016Review
- Site-Specific Genome Engineering in Human Pluripotent Stem Cells.International journal of molecular sciences · 2016Review
- Modeling psychiatric disorders with patient-derived iPSCs.Current opinion in neurobiology · 2016Review
- Human Embryonic Stem Cells: A Model for the Study of Neural Development and Neurological Diseases.Stem cells international · 2016Review
Corrections and comments
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Authors and funding
11 authors at 4 institutions in 1 country.
Funding
Abstract
Isogenic pluripotent stem cells are critical tools for studying human neurological diseases by allowing one to study the effects of a mutation in a fixed genetic background. Of particular interest are the spectrum of autism disorders, some of which are monogenic such as Timothy syndrome (TS); others are multigenic such as the microdeletion and microduplication syndromes of the 16p11.2 chromosomal locus. Here, we report engineered human embryonic stem cell (hESC) lines for modeling these two disorders using locus-specific endonucleases to increase the efficiency of homology-directed repair (HDR). We developed a system to: (1) computationally identify unique transcription activator-like effector nuclease (TALEN) binding sites in the genome using a new software program, TALENSeek, (2) assemble the TALEN genes by combining golden gate cloning with modified constructs from the FLASH protocol, and (3) test the TALEN pairs in an amplification-based HDR assay that is more sensitive than the typical non-homologous end joining assay. We applied these methods to identify, construct, and test TALENs that were used with HDR donors in hESCs to generate an isogenic TS cell line in a scarless manner and to model the 16p11.2 copy number disorder without modifying genomic loci with high sequence similarity.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.