Modeling and correction of protein conformational disease in iPSC-derived neurons through personalized base editing by Colin T. Konishi, Nancy Mulaiese, Tanvi Butola, Qinkun Zhang, Dana Kagan, Qiaoyan Yang, Mariel Pressler, Brooke G. Dirvin, Orrin Devinsky, Jayeeta Basu, Chengzu Long

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Targeted correction of megabase-scale CNTN6 duplication in induced pluripotent stem cells and impacts on gene expression by Maria Gridina, Polina Orlova, Oleg Serov

Subjects: “…CRISPR/Cas9-mediated genome editing…”
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Transitioning from wet lab to artificial intelligence: a systematic review of AI predictors in CRISPR by Ahtisham Fazeel Abbasi, Muhammad Nabeel Asim, Andreas Dengel

“…Abstract The revolutionary CRISPR-Cas9 system leverages a programmable guide RNA (gRNA) and Cas9 proteins to precisely cleave problematic regions within DNA sequences. …”
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Generation of Inducible CRISPRi and CRISPRa Human Stromal/Stem Cell Lines for Controlled Target Gene Transcription during Lineage Differentiation by Li Chen, Kaikai Shi, Weimin Qiu, Lars Aagaard, Moustapha Kassem

“…We infected hMSC with a CRISPR/Cas9 lentivirus system, with specific inducible dCas9-coupled transcription activator or repressor: dCas9-KRAB or dCas9-VP64, respectively, and established two hMSC lines (hMSC-CRISPRi and hMSC-CRISPRa) that can inhibit or activate gene expression, respectively. …”
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Nuclear-Targeting Delivery of CRISPRa System for Upregulation of β-Defensin against Virus Infection by Dexamethasone and Phenylalanine Dual-Modified Dendrimer by Mingxiang Zuo, Xiaoxia Li, Shuang Liu, Bin Chen, Du Cheng

“…The titer of vesicular stomatitis virus (VSV) in the cells treated with dCas9-sgRNA targeting β-defensin was reduced by about 100-fold compared to that of cells treated with dCas9-scramble sgRNA (dCas9-scr sgRNA). …”
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Manipulations with early mouse embryos for generation of genetically modified animals by A. N. Korablev, I. A. Serova, B. V. Skryabin

“…Until last time, the CRISPR/Cas9 system noticeably simplified the preparation of knockout or transgenic mice. …”
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Rational engineering of minimally immunogenic nucleases for gene therapy by Rumya Raghavan, Mirco J. Friedrich, Indigo King, Samuel Chau-Duy-Tam Vo, Daniel Strebinger, Blake Lash, Michael Kilian, Michael Platten, Rhiannon K. Macrae, Yifan Song, Lucas Nivon, Feng Zhang

“…In vivo editing of PCSK9 with SaCas9.Redi.1 is comparable in efficiency to wild-type SaCas9, but significantly reduces undesired immune responses. …”
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Principles and medical applications of gene editing by CRISPR / Cas by Rolando Sánchez Artigas, María Teresa Díaz Armas, Raisa Rodríguez Duque, Pedro Enrique Miguel Soca

“…A nuclear localization sequence allows CRISPR / Cas9 to enter the nucleus. CRISPR/Cas9 technology is an efficient tool for precise gene editing with great impact on scientific research.…”
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The Continuous Improvement of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)–CRISPR-Associated Protein System Has Led to Its Highly Efficient Application in... by Wanqing Tan, Zhiyuan Wang, Liezhao Liu

“…In this review, we systematically describe the Class II Cas proteins that have been used in plants, deactivated Cas9 (dCas9) and its role in transcriptional regulation, precision editing systems, Cas9 protein variants, as well as methods and examples of CRISPR–Cas systems targeting various regions with different breadths. …”
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Research Status of Clustered Regulary Interspaced Short Palindromic Repeats Technology in the Treatment of Human Papillomavirus (HPV) Infection Related Diseases by Minxue Tang MD

“…Background: CRISPR/Cas9 technology has rapidly advanced as a pivotal tool in cancer research, particularly in the precision targeting required for both detecting and treating malignancies. …”
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CRISPR-based epigenetic editing of Gad1 improves synaptic inhibition and cognitive behavior in a Tauopathy mouse model by Lei Wan, Ping Zhong, Pei Li, Yong Ren, Wei Wang, Mingjun Yu, Henry Y. Feng, Zhen Yan

“…Cells transfected with a fusion protein consisting of the nuclease-null dCas9 protein and the catalytic core of p300 (dCas9p300), as well as a guide RNA targeting Gad1 promoter (gRNAGad1), had significantly increased Gad1 mRNA expression and histone acetylation at Gad1 promoter. …”
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High frequency CCR5 editing in human hematopoietic stem progenitor cells protects xenograft mice from HIV infection by Daniel T. Claiborne, Zachary Detwiler, Steffen S. Docken, Todd D. Borland, Deborah Cromer, Amanda Simkhovich, Youdiil Ophinni, Ken Okawa, Timothy Bateson, Tao Chen, Wesley Hudson, Radiana Trifonova, Miles P. Davenport, Tony W. Ho, Christian L. Boutwell, Todd M. Allen

“…Our study demonstrates the feasibility of using CRISPR/Cas9/RNP to produce an HSPC transplant with high frequency CCR5 editing that is refractory to HIV replication. …”
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Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model by Toshio Miki, Ludivina Vazquez, Lisa Yanuaria, Omar Lopez, Irving M. Garcia, Kazuo Ohashi, Natalie S. Rodriguez

“…The Idua-deficient cells were competent to be reprogrammed to iPSCs, and pluripotency was maintained through CRISPR/CAS9-mediated gene correction. These results support the concept of ex vivo gene editing therapy using iPSC and CRISPR/Cas9 technologies for MPS-1 patients.…”
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Articular Cartilage Gene Expression after Coxofemoral Joint Luxation in the Dog by Korakot Nganvongpanit, Waranee Pradit, Siriwadee Chomdej

“…Ten mRNA, including nonapoptotic genes (AGG, COL2A1, MMP-3, HAS-1, HAS-2, and TIMP-1) and apoptotic genes (BAX, BCL-2, CAS-3, and CAS-9), were studied for their expression using real-time PCR. …”
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A comprehensive review of biological and genetic control approaches for leishmaniasis vector sand flies; emphasis towards promoting tools for integrated vector management. by Yasoda Kumari, Nayana Gunathilaka, Deepika Amarasinghe

“…Advances in genetic engineering technologies, particularly CRISPR/Cas9, sterile insect techniques, and gene drive insect modification, offer new avenues for precise and efficient sand fly management. …”
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Establishment of a CRISPR-Based Lentiviral Activation Library for Transcription Factor Screening in Porcine Cells by Yingjuan Liang, Xiaoxia Yao, Jingxin Han, Jinpeng Wang, Xiao Zhang, Donglin Zhao, Chaoqian Jiang, Lishuang Geng, Shihao Lv, Zhonghua Liu, Yanshuang Mu

“…A transcription-factor-scale activation library based on the clustered, regularly interspaced, short palindromic repeat (CRISPR)/CRISPR-associated endonuclease Cas9 (Cas9) system could facilitate the discovery and functional characterization of the transcription genes involved in a specific gene network. …”
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Islands of genomic stability in the face of genetically unstable metastatic cancer. by Kirsten Bowland, Jiaying Lai, Alyza Skaist, Yan Zhang, Selina Shiqing K Teh, Nicholas J Roberts, Elizabeth Thompson, Sarah J Wheelan, Ralph H Hruban, Rachel Karchin, Matthew H Bailey, Christine A Iacobuzio-Donahue, James R Eshleman

“…CRISPR-Cas9 PAM targets were determined by bioinformatic tumor-normal subtraction for each patient and verified in metastatic samples by high-depth capture-based sequencing.…”
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Navigating the frontier: Comprehensive insights into CRISPR technology advancements, delivery strategies, and ethical considerations in cancer research by Manisha S. Jain, K.S. Srikruthi, Prakash Goudanavar, N Raghavendra Naveen

“…Furthermore, the article explores the application of CRISPR/Cas9 gene editing to overcome cancer-mediated resistance and its potential role in drug delivery. …”
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Enhanced fetal hemoglobin production via dual-beneficial mutation editing of the HBG promoter in hematopoietic stem and progenitor cells for β-hemoglobinopathies by Prathibha Babu Chandraprabha, Manoj Kumar K. Azhagiri, Vigneshwaran Venkatesan, Wendy Magis, Kirti Prasad, Sevanthy Suresh, Aswin Anand Pai, Srujan Marepally, Alok Srivastava, Kumarasamypet Murugesan Mohankumar, David I. K. Martin, Saravanabhavan Thangavel

“…Conclusions Cas9 RNP-ssODN-based nucleotide conversion at the HBG promoter offers a promising gene therapy approach to ameliorate the phenotypes of β-thalassemia and SCD. …”
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Enhancers and genome conformation provide complex transcriptional control of a herpesviral gene by David W Morgens, Leah Gulyas, Xiaowen Mao, Alejandro Rivera-Madera, Annabelle S Souza, Britt A Glaunsinger

“…A parallel Cas9 nuclease screen indicated that three of these regions act as promoters of genes that regulate ORF68. …”
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