Article
Chemistry, Multidisciplinary
Yuyuan Wang, Pawan K. Shahi, Xiuxiu Wang, Ruosen Xie, Yi Zhao, Min Wu, Seth Roge, Bikash R. Pattnaik, Shaoqin Gong
Summary: A stimulus-responsive silica nanoparticle has been developed for efficient delivery of nucleic acids and CRISPR genome editors, showing great potential for applications in gene therapy and genome editing.
JOURNAL OF CONTROLLED RELEASE
(2021)
Article
Chemistry, Physical
Juhee Lee, Yoo Kyung Kang, Eonju Oh, Juhee Jeong, San Hae Im, Duk Ki Kim, Haeshin Lee, Sang-Gyu Kim, Keehoon Jung, Hyun Jung Chung
Summary: The study presents a cancer gene therapy strategy based on NanoRNP that efficiently blocks the PD-L1 immune checkpoint and induces an antitumor effect in vivo without the need for combination therapy. In vivo results demonstrate that NanoRNP can induce indels in target cells at high frequencies, significantly suppressing tumor growth.
CHEMISTRY OF MATERIALS
(2022)
Review
Biochemistry & Molecular Biology
Dengyun Nie, Ting Guo, Miao Yue, Wenya Li, Xinyu Zong, Yinxing Zhu, Junxing Huang, Mei Lin
Summary: CRISPR/Cas9 technology is a powerful gene editing tool with great potential in oncology. However, its efficiency and safety issues restrict its widespread application. Developing a suitable delivery method, such as nanoparticles, is urgently needed.
Article
Biotechnology & Applied Microbiology
Rajveer Singh, Shivani Chandel, Arijit Ghosh, Anupam Gautam, Daniel H. Huson, V. Ravichandiran, Dipanjan Ghosh
Summary: In the past two decades, yeast has been widely used as a cost-effective biological tool for producing small molecules and biofuels. The application of CRISPR-Cas techniques in yeast genetic and metabolic engineering has revolutionized the field, particularly in targeted chromosomal integration using synthetic donor constructs. This study presents a highly efficient strategy for targeted chromosomal integration and in-frame expression of foreign genes in the yeast genome using CRISPR-Cas9 and homology-dependent recombination (HDR). The optimized methods achieved a 74% efficiency in integrating small constructs at multiple target sites. The study also demonstrated that as few as 15 base pairs of microhomology are sufficient for targeted knock-in with minimal construct concentration. Whole-genome sequencing confirmed the absence of off-target effects. This protocol provides a comprehensive and streamlined approach for integrating essential genes into the yeast genome for synthetic biology and industrial purposes.
Review
Medicine, Research & Experimental
Asma Ghaemi, Elnaz Bagheri, Khalil Abnous, Seyed Mohammad Taghdisi, Mohammad Ramezani, Mona Alibolandi
Summary: The prokaryotic CRISPR-Cas systems serve as a revolutionary genome editing tool for modifying, visualizing, and identifying DNA and RNA sequences in live cells of various species. Besides genome editing, the DNA-targeting modifications by CRISPR-Cas offer opportunities for diagnosis, therapy, and genetic disorders investigation. Preclinical studies and CRISPR-Cas9-based therapeutics may encounter challenges, but there is potential for its development in cancer therapy.
Review
Chemistry, Multidisciplinary
Deepak Kumar Sahel, Lalitkumar K. Vora, Aishwarya Saraswat, Saurabh Sharma, Jasmin Monpara, Anisha A. D'Souza, Deepakkumar Mishra, Kamatham Pushpa Tryphena, Satoru Kawakita, Shahid Khan, Mohd Azhar, Dharmendra Kumar Khatri, Ketan Patel, Raghu Raj Singh Thakur
Summary: CRISPR and Cas9, initially discovered as an immune system in bacteria and archaea, are now widely used as biotechnological tools for genetic and nongenetic disease treatment. Clinical trials mainly adopt ex vivo strategies, but in vivo delivery methods for CRISPR components are still under preclinical surveillance.
Article
Chemistry, Multidisciplinary
Chang Liu, Xiaoyu Xu, Oliver Koivisto, Wenhui Zhou, Guillaume Jacquemet, Jessica M. Rosenholm, Hongbo Zhang
Summary: Appropriate tuning of robust artificial coatings can enhance intracellular delivery and preserve biological functions. Synthesizing controllable nanostructures by encapsulating CRISPR/Cas9 plasmids into metal-organic frameworks can dramatically impact the biological features of the plasmids. The MOF-coated nanostructures have excellent loading capability, pH-responsive release, and protection from enzymatic degradation.
Review
Pharmacology & Pharmacy
Xin-Chi Jiang, Tianyuan Zhang, Jian-Qing Gao
Summary: This review describes the preparation process of exosomes and biomimetic vesicles, and summarizes the mechanism of loading and delivery of nucleic acids or gene editing systems. It provides a comprehensive overview of the techniques employed for preparing the targeting crossover vesicles based on their cellular uptake and targeting mechanism. The challenges and clinical applications of vesicles are also discussed.
ADVANCED DRUG DELIVERY REVIEWS
(2022)
Article
Multidisciplinary Sciences
Min Qiu, Zachary Glass, Jinjin Chen, Mary Haas, Xin Jin, Xuewei Zhao, Xuehui Rui, Zhongfeng Ye, Yamin Li, Feng Zhang, Qiaobing Xu
Summary: Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are linked to reduced blood lipid levels, making it a potential therapeutic target for lipid metabolism disorders. A lipid nanoparticle delivery system successfully edited Angptl3 gene in vivo, leading to significant reductions in lipid levels without off-target effects or toxicity, highlighting the potential of this platform for safe and effective gene editing therapies.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
(2021)
Article
Endocrinology & Metabolism
Divya Ramachandran, Kusum Sharma, Vishal Saxena, Niepukolie Nipu, Dinusha C. Rajapaksha, Jan A. Mennigen
Summary: The nonapeptide vasotocin/vasopressin is highly conserved and plays a role as neuromodulator and signaling molecule in vertebrates. Pharmacological studies in several teleost fishes suggest that vasotocin has sex and species-specific reproductive roles. However, comprehensive studies investigating the behavioral and physiological consequences of genetic ablation of vasotocin in a genetically tractable fish model, such as the zebrafish, are currently lacking.
FRONTIERS IN ENDOCRINOLOGY
(2023)
Article
Chemistry, Multidisciplinary
Xiaoyu Xu, Chang Liu, Shengyi Wang, Ermei Makila, Jiali Wang, Oliver Koivisto, Junnian Zhou, Jessica M. Rosenholm, Yilai Shu, Hongbo Zhang
Summary: In this study, a microfluidic-assisted metal-organic framework (MOF) based biomineralization strategy was developed for the efficient and remote control delivery of CRISPR/Cas9 RNP gene editing. The strategy involved growing thermo-responsive EuMOFs onto a photothermal template Prussian blue (PB) and loading RNP during MOFs crystallization in microfluidic channels. The resulting nanocarrier showed high loading efficiency, protection of RNP, and NIR-stimulated release capacity. Upon laser exposure, the nanocarrier demonstrated effective endosomal escape and precise gene knockout. The gene-editing activity could be programmed by tuning exposure times, providing a more controllable editing modality compared to the control group without laser irradiation.
Article
Medicine, Research & Experimental
Eleonora Maino, Daria Wojtal, Sonia L. Evagelou, Aiman Farheen, Tatianna W. Y. Wong, Kyle Lindsay, Ori Scott, Samar Z. Rizvi, Elzbieta Hyatt, Matthew Rok, Shagana Visuvanathan, Amanda Chiodo, Michelle Schneeweiss, Evgueni A. Ivakine, Ronald D. Cohn
Summary: Tandem duplication mutations are increasingly being recognized as a direct cause of rare heritable diseases, but the lack of suitable animal models has hindered research and therapeutic development. A novel mouse model with a multi-exonic tandem duplication in the Dmd gene was successfully corrected using a CRISPR/Cas9 approach, leading to restoration of full-length dystrophin expression and improvements in histopathological and clinical phenotypes. This study highlights the potential of CRISPR/Cas9 as a powerful tool for accurately modeling and treating tandem duplication mutations.
EMBO MOLECULAR MEDICINE
(2021)
Article
Medicine, Research & Experimental
Qingxia Zuo, Wanqing Xu, Yanbin Wan, Dongyan Feng, Changsheng He, Cailing Lin, Dongchao Huang, Feng Chen, Liya Han, Qi Sun, Dong Chen, Hongli Du, Lizhen Huang
Summary: CRISPR/Cas9 was used to generate a CYP3A4-T2A-luciferase knock-in HepaRG subclone, and optimized differentiation procedures were found to promote differentiation and improve the expression of hepatocyte-specific genes.
BIOMEDICINE & PHARMACOTHERAPY
(2022)
Article
Biotechnology & Applied Microbiology
Andreas Braae Holmgaard, Anne Louise Askou, Emilie Grarup Jensen, Sidsel Alsing, Rasmus O. Bak, Jacob Giehm Mikkelsen, Thomas J. Corydon
Summary: This study investigates the therapeutic effect of retinal gene therapy using CRISPR/Cas9-mediated genome editing and knockout applications. By utilizing RNPs composed of msgRNAs and SpCas9, efficacy can be enhanced to achieve targeted gene knockout. Moreover, lipoplex delivery of a mixture of RNP and mRNA has been shown to achieve efficient knockout.
Article
Multidisciplinary Sciences
Amita Tiyaboonchai, Anne Vonada, Jeffrey Posey, Carl Pelz, Leslie Wakefield, Markus Grompe
Summary: This study uses self-cleaving gRNAs to create drug-selectable gene editing events in specific hepatocyte loci, resulting in expanded gene-edited cells and therapeutic levels of gene expression.
NATURE COMMUNICATIONS
(2022)
Article
Biotechnology & Applied Microbiology
Maurizio Buggio, Christopher Towe, Anand Annan, Sergey Kaliberov, Zhi Hong Lu, Calvin Stephens, Jeffrey M. Arbeit, David T. Curiel
JOURNAL OF GENE MEDICINE
(2016)
Article
Biochemistry & Molecular Biology
Gareth Highnam, Christopher Franck, Andy Martin, Calvin Stephens, Ashwin Puthige, David Mittelman
NUCLEIC ACIDS RESEARCH
(2013)
Article
Chemistry, Multidisciplinary
Calvin J. Stephens, Elvin J. Lauron, Elena Kashentseva, Zhi Hong Lu, Wayne M. Yokoyama, David T. Curiel
JOURNAL OF CONTROLLED RELEASE
(2019)
Article
Biotechnology & Applied Microbiology
Shinnosuke Tsuji, Calvin J. Stephens, Giulia Bortolussi, Feijie Zhang, Gabriele Baj, Hagoon Jang, Gustavo de Alencastro, Andres F. Muro, Katja Pekrun, Mark A. Kay
Summary: The use of ribonucleotide reductase inhibitors can substantially enhance the efficiency of adeno-associated virus-mediated homologous recombination-based gene therapy. These findings suggest that clinically approved inhibitors, such as fludarabine, can be used to potentiate gene editing therapies.
NATURE BIOTECHNOLOGY
(2022)
Meeting Abstract
Biotechnology & Applied Microbiology
Katja Pekrun, Calvin J. Stephens, Feijie Zhang, Lauren Kelly, Natacha Le Moan, Alice F. Tarantal, Grant E. Blouse, Mark A. Kay
Meeting Abstract
Biotechnology & Applied Microbiology
Calvin J. Stephens, Katja Pekrun, Jianpeng Xu, Shinnosuke Tsuji, Yuqing Jing, Francesco Puzzo, Feijie Zhang, Mark A. Kay
Meeting Abstract
Biotechnology & Applied Microbiology
Hagoon Jang, Kirk Chu, Feijie Zhang, Calvin Stephens, Mark A. Kay
Meeting Abstract
Biotechnology & Applied Microbiology
Shinnosuke Tsuji, Calvin J. Stephens, Giulia Bortolussi, Katja Pekrun, Feijie Zhang, Gustavo de Alencastro, Gabriele Baj, Andres F. Muro, Mark A. Kay
Meeting Abstract
Biotechnology & Applied Microbiology
Calvin J. Stephens, Katja Pekrun, Shinnosuke Tsuji, Feijie Zhang, Mark A. Kay
Meeting Abstract
Biotechnology & Applied Microbiology
Shinnoskuke Tsuji, Calvin J. Stephens, Katja Pekrun, Feijie Zhang, Arjun Zhang, Mark A. Kay
Meeting Abstract
Biotechnology & Applied Microbiology
C. J. Stephens, E. Kashentseva, W. Everett, L. Kaliberova, D. T. Curiel
HUMAN GENE THERAPY
(2017)
Meeting Abstract
Biotechnology & Applied Microbiology
Calvin Stephens, Elena Kashentseva, William Everett, Lyudmila Kaliberova, David T. Curiel