Search

Your search keyword '"crispr/Cas9"' showing total 19,924 results
Start Over Descriptor "crispr/Cas9" Publication Year Range Last 50 years

Refine your results

19,924 results on '"crispr/Cas9"'

23. A tripartite circRNA/mRNA/miRNA interaction regulates glutamatergic signaling in the mouse brain

Author

Silenzi, Valentina, D’Ambra, Eleonora, Santini, Tiziana, D’Uva, Sara, Setti, Adriano, Salvi, Nicolò, Nicoletti, Carmine, Scarfò, Rebecca, Cordella, Federica, Mongiardi, Brunella, Cavezza, Diletta, Liessi, Nara, Ferrucci, Laura, Ragozzino, Davide, Armirotti, Andrea, Di Angelantonio, Silvia, De Leonibus, Elvira, Bozzoni, Irene, and Morlando, Mariangela

Published
2024
Full Text
View/download PDF

44. A temperature-sensitive and less immunogenic Sendai virus for efficient gene editing.

Author

Stevens, Christian, Carmichael, Jillian, Watkinson, Ruth, Kowdle, Shreyas, Reis, Rebecca, Hamane, Kory, Jang, Jason, Park, Arnold, Pernet, Olivier, Khamaikawin, Wannisa, Hong, Patrick, Thibault, Patricia, Gowlikar, Aditya, An, Dong, and Lee, Benhur

Subjects
CCR5, CRISPR/Cas9, HIV, Paramyxoviridae, Sendai virus, gene editing, hematopoietic stem and progenitor cells, monocytes, viral vector, Gene Editing, Humans, Sendai virus, Receptors, CCR5, Hematopoietic Stem Cells, Genetic Vectors, CRISPR-Cas Systems, Temperature, HIV-1, Transduction, Genetic, HIV Infections, Monocytes
Abstract

UNLABELLED: The therapeutic potential of gene editing technologies hinges on the development of safe and effective delivery methods. In this study, we developed a temperature-sensitive and less immunogenic Sendai virus (ts SeV) as a novel delivery vector for CRISPR-Cas9 and for efficient gene editing in sensitive human cell types with limited induction of an innate immune response. ts SeV demonstrates high transduction efficiency in human CD34+ hematopoietic stem and progenitor cells (HSPCs) including transduction of the CD34+/CD38-/CD45RA-/CD90+(Thy1+)/CD49fhigh stem cell enriched subpopulation. The frequency of CCR5 editing exceeded 90% and bi-allelic CCR5 editing exceeded 70% resulting in significant inhibition of HIV-1 infection in primary human CD14+ monocytes. These results demonstrate the potential of the ts SeV platform as a safe, efficient, and flexible addition to the current gene-editing tool delivery methods, which may help further expand the possibilities in personalized medicine and the treatment of genetic disorders. IMPORTANCE: Gene editing has the potential to be a powerful tool for the treatment of human diseases including HIV, β-thalassemias, and sickle cell disease. Recent advances have begun to overcome one of the major limiting factors of this technology, namely delivery of the CRISPR-Cas9 gene editing machinery, by utilizing viral vectors. However, gene editing therapies have yet to be implemented due to inherent risks associated with the DNA viral vectors typically used for delivery. As an alternative strategy, we have developed an RNA-based Sendai virus CRISPR-Cas9 delivery vector that does not integrate into the genome, is temperature sensitive, and does not induce a significant host interferon response. This recombinant SeV successfully delivered CRISPR-Cas9 in primary human CD14+ monocytes ex vivo resulting in a high level of CCR5 editing and inhibition of HIV infection.

Published
2024

45. Widespread Gene Editing in the Brain via In Utero Delivery of mRNA Using Acid-Degradable Lipid Nanoparticles

Author

Gao, Kewa, Han, Hesong, Cranick, Matileen G, Zhao, Sheng, Xu, Shanxiu, Yin, Boyan, Song, Hengyue, Hu, Yibo, Clarke, Maria T, Wang, David, Wong, Jessica M, Zhao, Zehua, Burgstone, Benjamin W, Farmer, Diana L, Murthy, Niren, and Wang, Aijun

Subjects
Medical Biotechnology, Biomedical and Clinical Sciences, Neurosciences, Intellectual and Developmental Disabilities (IDD), Pediatric, Bioengineering, Nanotechnology, Biotechnology, Gene Therapy, Stem Cell Research, Mental Health, Brain Disorders, Genetics, Perinatal Period - Conditions Originating in Perinatal Period, 2.1 Biological and endogenous factors, 5.1 Pharmaceuticals, 5.2 Cellular and gene therapies, Reproductive health and childbirth, Neurological, Animals, Mice, Brain, RNA, Messenger, Nanoparticles, Female, Lipids, Gene Editing, Pregnancy, Polyethylene Glycols, Liposomes, in utero, gene editing, mRNA delivery, nanoparticles, CRISPR/Cas9, CNS disorder, Nanoscience & Nanotechnology
Abstract

In utero gene editing with mRNA-based therapeutics has the potential to revolutionize the treatment of neurodevelopmental disorders. However, a critical bottleneck in clinical application has been the lack of mRNA delivery vehicles that can efficiently transfect cells in the brain. In this report, we demonstrate that in utero intracerebroventricular (ICV) injection of densely PEGylated lipid nanoparticles (ADP-LNPs) containing an acid-degradable PEG-lipid can safely and effectively deliver mRNA for gene editing enzymes to the fetal mouse brain, resulting in successful transfection and editing of brain cells. ADP-LNPs containing Cre mRNA transfected 30% of the fetal brain cells in Ai9 mice and had no detectable adverse effects on fetal development and postnatal growth. In addition, ADP-LNPs efficiently transfected neural stem and progenitor cells in Ai9 mice with Cre mRNA, which subsequently proliferated and caused over 40% of the cortical neurons and 60% of the hippocampal neurons to be edited in treated mice 10 weeks after birth. Furthermore, using Angelman syndrome, a paradigmatic neurodevelopmental disorder, as a disease model, we demonstrate that ADP-LNPs carrying Cas9 mRNA and gRNA induced indels in 21% of brain cells within 7 days postpartum, underscoring the precision and potential of this approach. These findings demonstrate that LNP/mRNA complexes have the potential to be a transformative tool for in utero treatment of neurodevelopmental disorders and set the stage for a frontier in treating neurodevelopmental disorders that focuses on curing genetic diseases before birth.

Published
2024

46. Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene

Author

Cardinali, Beatrice, Provenzano, Claudia, Izzo, Mariapaola, Voellenkle, Christine, Battistini, Jonathan, Strimpakos, Georgios, Golini, Elisabetta, Mandillo, Silvia, Scavizzi, Ferdinando, Raspa, Marcello, Perfetti, Alessandra, Baci, Denisa, Lazarevic, Dejan, Garcia-Manteiga, Jose Manuel, Gourdon, Geneviève, Martelli, Fabio, and Falcone, Germana

Published
2022
Full Text
View/download PDF

47. Application of CRISPR/Cas9 in the Improvement of Industrially Important Crops

Author

Ghosh, Gourab, Padaria, Jasdeep Chatrath, Amer, Mourad, Series Editor, Pollice, Fabio, Editorial Board Member, Darko, Amos, Editorial Board Member, Ujang, Muhamad Uznir, Editorial Board Member, Rodrigo-Comino, Jesús, Editorial Board Member, El Kaftangui, Mohamed, Editorial Board Member, Battisti, Alessandra, Editorial Board Member, Albatayneh, Aiman, Editorial Board Member, Turan, Veysel, Editorial Board Member, Doronzo, Domenico M., Editorial Board Member, Morsy, Alaa M., Editorial Board Member, Yehia, Moustafa, Editorial Board Member, Di Stefano, Elisabetta, Editorial Board Member, Salih, Gasim Hayder Ahmed, Editorial Board Member, Michel, Mina, Editorial Board Member, Vishwakarma, Vinita, Editorial Board Member, Mortada, Ashraf, Editorial Board Member, Mehmet, Alkan, Editorial Board Member, Jat, Mahesh Kumar, Editorial Board Member, Gallo, Paola, Editorial Board Member, Aref, M. M. El, Editorial Board Member, Hamimi, Zakaria, Editorial Board Member, Elewa, Ahmed Kalid, Editorial Board Member, Trapani, Ferdinando, Editorial Board Member, Alberti, Francesco, Editorial Board Member, Maarouf, Ibrahim, Editorial Board Member, Soliman, Akram M., Editorial Board Member, and Kumar, Nitish, editor

Published
2025
Full Text
View/download PDF

Catalog

Books, media, physical & digital resources
See catalog results