ArticleScientific reports2016
Rapid and efficient CRISPR/Cas9 gene inactivation in human neurons during human pluripotent stem cell differentiation and direct reprogramming.
Article in Scientific reports, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 27 papers.
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Who cites it
27 citing papers in PubMed, 43 citations in OpenAlex.
- Targeted AAV gene therapy for neuroblastoma via direct capsid-antibody coupling.EMBO molecular medicine · 2026Article
- Revisiting Pluripotency Acquisition: A Unified Framework for Reprogramming Strategies.Development, growth & differentiation · 2026Review
- An electrophysiological and proteomics roadmap for human induced glutamatergic neurons: fine-tuning of culture conditions for pathophysiological studies.Cell death discovery · 2026Article
- Human pluripotent stem cell engineering with CRISPR-Cas9 for Parkinson's disease.Experimental & molecular medicine · 2026Review
- New AAV9 engineered variants with enhanced neurotropism and reduced liver off-targeting in mice and marmosets.iScience · 2024Article
- Cell Reprogramming and Differentiation Utilizing Messenger RNA for Regenerative Medicine.Journal of developmental biology · 2023Review
- Somatic Cell Reprogramming for Nervous System Diseases: Techniques, Mechanisms, Potential Applications, and Challenges.Brain sciences · 2023Review
- Article
- Updated Perspectives on Direct Vascular Cellular Reprogramming and Their Potential Applications in Tissue Engineered Vascular Grafts.Journal of functional biomaterials · 2022Review
- Human-Induced Pluripotent Stem Cell Technology: Toward the Future of Personalized Psychiatry.Journal of personalized medicine · 2022Review
- Application of Small Molecules in the Central Nervous System Direct Neuronal Reprogramming.Frontiers in bioengineering and biotechnology · 2022Review
- Frataxin gene editing rescues Friedreich's ataxia pathology in dorsal root ganglia organoid-derived sensory neurons.Nature communications · 2020Article
- In Vitro Differentiated Human Stem Cell-Derived Neurons Reproduce Synaptic Synchronicity Arising during Neurodevelopment.Stem cell reports · 2020Article
- Whole brain delivery of an instability-proneeLife · 2020Article
- Prospects of Directly Reprogrammed Adult Human Neurons for Neurodegenerative Disease Modeling and Drug Discovery: iN vs. iPSCs Models.Frontiers in neuroscience · 2020Review
- Advances in Transcription Factors Related to Neuroglial Cell Reprogramming.Translational neuroscience · 2020Article
- Direct cell-fate conversion of somatic cells: Toward regenerative medicine and industries.Proceedings of the Japan Academy. Series B, Physical and biological sciences · 2020Review
- Novel Glial Cell Functions: Extensive Potency, Stem Cell-Like Properties, and Participation in Regeneration and Transdifferentiation.Frontiers in cell and developmental biology · 2020Review
- Transcription Factor-Based Fate Specification and Forward Programming for Neural Regeneration.Frontiers in cellular neuroscience · 2020Review
- Stem Cell Modeling of Neuroferritinopathy Reveals Iron as a Determinant of Senescence and Ferroptosis during Neuronal Aging.Stem cell reports · 2019Article
Corrections and comments
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Authors and funding
12 authors at 3 institutions in 1 country.
Funding
Abstract
The CRISPR/Cas9 system is a rapid and customizable tool for gene editing in mammalian cells. In particular, this approach has widely opened new opportunities for genetic studies in neurological disease. Human neurons can be differentiated in vitro from hPSC (human Pluripotent Stem Cells), hNPCs (human Neural Precursor Cells) or even directly reprogrammed from fibroblasts. Here, we described a new platform which enables, rapid and efficient CRISPR/Cas9-mediated genome targeting simultaneously with three different paradigms for in vitro generation of neurons. This system was employed to inactivate two genes associated with neurological disorder (TSC2 and KCNQ2) and achieved up to 85% efficiency of gene targeting in the differentiated cells. In particular, we devised a protocol that, combining the expression of the CRISPR components with neurogenic factors, generated functional human neurons highly enriched for the desired genome modification in only 5 weeks. This new approach is easy, fast and that does not require the generation of stable isogenic clones, practice that is time consuming and for some genes not feasible.
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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.