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Epigenetics in Multiple Sclerosis

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Methodologies and Challenges for CRISPR/Cas9 Mediated Genome Editing of the Mammalian Brain

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Revisiting gene delivery to the brain: silencing and editing

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Transcriptional activation with Cas9 activator nanocomplexes rescues Alzheimer's disease pathology

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CRISPR-Based Approaches for the High-Throughput Characterization of Long Non-Coding RNAs

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A Cre-Dependent CRISPR/dCas9 System for Gene Expression Regulation in Neurons

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Dynamic properties of transcriptional condensates modulate CRISPRa-mediated gene activation

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RGS Proteins as Critical Regulators of Motor Function and Their Implications in Parkinson’s Disease

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Control of CRK-RAC1 activity by the miR-1/206/133 miRNA family is essential for neuromuscular junction function

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CRISPR-mediated activation of autism gene Itgb3 restores cortical network excitability via mGluR5 signaling

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Gene Modulation with CRISPR-based Tools in Human iPSC-Cardiomyocytes

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Current therapies for osteoarthritis and prospects of CRISPR-based genome, epigenome, and RNA editing in osteoarthritis treatment

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CRISPR activation screens identify the SWI/SNF ATPases as suppressors of ferroptosis

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CRISPR: A Screener’s Guide

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APOE4/4 is linked to damaging lipid droplets in Alzheimer’s microglia

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Genetics and epigenetics of allergy

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Programmable Transcriptional Modulation with a Structured RNA-Mediated CRISPR-dCas9 Complex

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CRISPR for neuroscientists

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Optimized dCas9 programmable transcriptional activators for plants

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Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

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CRISPR and transposon in vivo screens for cancer drivers and therapeutic targets

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Application of CRISPR/Cas Genomic Editing Tools for HIV Therapy: Toward Precise Modifications and Multilevel Protection

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RNA variant assessment using transactivation and transdifferentiation

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Specific multivalent molecules boost CRISPR-mediated transcriptional activation

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Applications of CRISPR-Cas Technologies to Proteomics

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CRISPR activation and interference as investigative tools in the cardiovascular system

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Distinct roles of Bdnf I and Bdnf IV transcript variant expression in hippocampal neurons

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Recent advances in neuroepigenetic editing

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Genetically Engineering the Nervous System with CRISPR-Cas

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Application of Various Delivery Methods for CRISPR/dCas9

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DNA methylation occurring in Cre‐expressing cells inhibits loxP recombination and silences loxP‐sandwiched genes

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Endogenous promoter-driven sgRNA for monitoring the expression of low-abundance transcripts and lncRNAs

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Protein Scaffold‐Based Multimerization of Soluble ACE2 Efficiently Blocks SARS‐CoV‐2 Infection In Vitro and In Vivo

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Modulating cancer stemness provides luminal a breast cancer cells with HER2 positive-like features

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New role of astrocytes in neuroprotective mechanisms after ischemic stroke

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Acoustically targeted noninvasive gene therapy in large brain volumes

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Massively parallel in vivo Perturb-seq screening

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Integration of CRISPR-engineering and hiPSC-based models of psychiatric genomics

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Use of Customizable Nucleases for Gene Editing and Other Novel Applications

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Identification of stress-induced epigenetic methylation onto dopamine D2 gene and neurological and behavioral consequences

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Elevation of master autophagy regulator Tfeb in osteoblast lineage cells increases bone mass and strength

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Orthogonal transcriptional modulation and gene editing using multiple CRISPR-Cas systems

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Role of long non-coding RNAs in neurofibromatosis and Schwannomatosis: pathogenesis and therapeutic potential

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Regulation of Gene Expression and the Elucidative Role of CRISPR-Based Epigenetic Modifiers and CRISPR-Induced Chromosome Conformational Changes

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Epigenetic pharmacotherapy for substance use disorder

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Lentiviral CRISPR/Cas9-Mediated Genome Editing for the Study of Hematopoietic Cells in Disease Models

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Genetic therapies and potential therapeutic applications of CRISPR activators in the eye

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Diversification of the CRISPR Toolbox: Applications of CRISPR-Cas Systems Beyond Genome Editing

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Develop an efficient and specific AAV-based labeling system for Muller glia in mice

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Applications of CRISPR technology in cellular immunotherapy

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Genetic and epigenetic editing in nervous system

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Discovering Essential Multiple Gene Effects Through Large Scale Optimization: An Application to Human Cancer Metabolism

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MicroRNA mechanisms instructing Purkinje cell specification

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Application of CRISPR for In Vivo Mouse Cancer Studies

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Contribution of structural accessibility to the cooperative relationship of TF-lncRNA in myopia

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Quantitative Detection and Real-Time Monitoring of Endogenous mRNA at the Single Live Cell Level Using a Ratiometric Molecular Beacon

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Neuronal Cell-type Engineering by Transcriptional Activation

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Genetic correction strategies for Duchenne muscular dystrophy and their impact on the heart

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CRISPR-Based Transcriptional Regulation: Technologies, Applications, and Future Directions

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CRISPR-broad: combined design of multi-targeting gRNAs and broad, multiplex target finding

Veluchamy, A., Teles, K. A., & Fischle, W. (2023) · Scientific Reports · doi:10.1038/s41598-023-46212-x · Cited by 10

Direct reprogramming of fibroblasts into functional hepatocytes via CRISPRa activation of endogenous Gata4 and Foxa3

Li, J., Li, R., Bai, X., Zhang, W., Nie, Y., & Hu, S. (2024) · Chinese Medical Journal · doi:10.1097/cm9.0000000000003088 · Cited by 10

CRISPR applications in microbial World: Assessing the opportunities and challenges

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CRISPR/Cas: a potential gene-editing tool in the nervous system

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Dead Cas(t) light on new life: CRISPRa-mediated reprogramming of somatic cells into neurons

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Gene activation guided by nascent RNA-bound transcription factors

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Direct Cell Reprogramming and Phenotypic Conversion: An Analysis of Experimental Attempts to Transform Astrocytes into Neurons in Adult Animals

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Application of CRISPR-Cas systems in neuroscience

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Generation of Leydig-like cells: approaches, characterization, and challenges

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Recent advances of genome editing and related technologies in China

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Gene regulatory and gene editing tools and their applications for retinal diseases and neuroprotection: From proof-of-concept to clinical trial

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Novel methods for the generation of genetically engineered animal models

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Enrichment of transgene integrations by transient CRISPR activation of a silent reporter gene

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CRISPR-Mediated Activation of αV Integrin Subtypes Promotes Neuronal Differentiation of Neuroblastoma Neuro2a Cells

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Harnessing CRISPR potential for intervertebral disc regeneration strategies

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Genetics of Dravet Syndrome and its Targeted Therapy by Nanomedicine: A Roadmap for Future Treatment of Drug Resistant Seizures

Ikram, M. D., & Rasheed, S. (2022) · Current Molecular Pharmacology · doi:10.2174/1874467215666220819143105 · Cited by 8

Eomesodermin spatiotemporally orchestrates the early and late stages of NK cell development by targeting KLF2 and T-bet, respectively

He, J., Chen, D., Xiong, W., Hou, X., Quan, Y., Yang, M., & Dong, Z. (2024) · Cellular and Molecular Immunology · doi:10.1038/s41423-024-01164-8 · Cited by 8

Transgenerational Epigenetic DNA Methylation Editing and Human Disease

Tompkins, J. D. (2023) · Biomolecules · doi:10.3390/biom13121684 · Cited by 13

Leveraging CRISPR activation for rapid assessment of gene editing products in human pluripotent stem cells

Wu, Y., Zhong, A., Evangelisti, A., Sidharta, M., Huangfu, D., Studer, L., & Zhou, T. (2025) · Stem Cell Reports · doi:10.1016/j.stemcr.2025.102499 · Cited by 7

Novel drug-inducible CRISPRa/i systems for rapid and reversible manipulation of gene transcription

Sui, M., Zhou, M., Cui, M., Liu, H., Zhang, X., Hu, N., Li, Y., Wang, B., Yang, G., Gui, P., et al. (2025) · Cellular and Molecular Life Sciences · doi:10.1007/s00018-025-05786-7 · Cited by 7

Strategies and mechanisms of precision genome engineering: From gene editing to genome writing

Huang, K., Tian, J., Zhao, W., Zhang, S., Xiang, L., Yang, M., Ls, L., Huang, Y., Zhang, N., Sun, L., et al. (2026) · iMetaOmics. · doi:10.1002/imo2.70115 · Cited by 8

How can preclinical mouse models be used to gain insight into prefrontal cortex dysfunction in obsessive-compulsive disorder?

Manning, E. E., & Ahmari, S. E. (2018) · Brain and Neuroscience Advances · doi:10.1177/2398212818783896 · Cited by 8

Heart Enhancers: Development and Disease Control at a Distance

Yuan, X., Scott, I. C., & Wilson, M. D. (2021) · Frontiers in Genetics · doi:10.3389/fgene.2021.642975 · Cited by 7

Correction of β3 integrin haplo-insufficiency by CRISPRa normalizes cortical network activityPreprint

Jaudon, F., Thalhammer, A., & Cingolani, L. A. (2019) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/664706 · Cited by 7

Next-generation direct reprogramming

Keshri, R., Detraux, D., Phal, A., McCurdy, C., Jhajharia, S., Chan, T. C., Mathieu, J., & Ruohola‐Baker, H. (2024) · Frontiers in Cell and Developmental Biology · doi:10.3389/fcell.2024.1343106 · Cited by 7

Development of artificial transcription factors and their applications in cell reprograming, genetic screen, and disease treatment

Sang, Y., Xu, L., & Bao, Z. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.10.029 · Cited by 9

Cardiomyocyte engineering: The meeting point of transcription factors, signaling networks, metabolism and function

Piven, O. O., Vaičiulevičiūtė, R., Bernotienė, E., & Dobrzyń, P. (2025) · Acta Physiologica · doi:10.1111/apha.14271 · Cited by 6

From qualitative to quantitative: the state of the art and challenges for plant synthetic biology

Tian, C., Li, J., & Wang, Y. (2023) · Quantitative Biology · doi:10.15302/j-qb-022-0326 · Cited by 5

A Neuron-Optimized CRISPR/dCas9 Activation System for Robust and Specific Gene Regulation

Savell, K. E., Bach, S. V., Zipperly, M. E., Revanna, J. S., Goska, N. A., Tuscher, J. J., Duke, C. G., Sultan, F. A., Burke, J. N., Williams, D., et al. (2019) · eNeuro · doi:10.1523/eneuro.0495-18.2019 · Cited by 137

Study is needed of visual field defects associated with any long term antiepileptic drug

Rao, G. P., Ah-Fat, F. G., Kyle, G., Leach, J. P., Chadwick, D. W., & Batterbury, M. (1998) · BMJ · doi:10.1136/bmj.317.7152.206 · Cited by 18

Toehold-Based CRISPR-dCas9 Transcriptional Activation Platform for Spatiotemporally Controllable Gene Therapy in Tumor and Diabetic Mouse Models

Hu, C., Shi, X., Guo, B., Yang, Z., Zhou, J. H. S., & Wang, F. (2025) · ACS Nano · doi:10.1021/acsnano.5c01078 · Cited by 13

Transgenic mice for in vivo epigenome editing with CRISPR-based systemsPreprint

Gemberling, M. P., Siklenka, K., Rodriguez, E., Eisinger, K. R. T., Barrera, A., Liu, F., Kantor, A., Li, L., Cigliola, V., Hazlett, M. F., et al. (2021) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2021.03.08.434430 · Cited by 3

One-Step piggyBac Transposon-Based CRISPR/Cas9 Activation of Multiple Genes

Li, S., Zhang, A., Xue, H., Li, D., & Liu, Y. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.06.007 · Cited by 35