Clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) technology enables targeted gene editing, but cancer gene therapy with this approach requires improvements to enable safe and efficient delivery of CRISPR/Cas9 to tumors. We developed and evaluated a self-assembled liposome to selectively deliver CRISPR/Cas9 to cancer tissues. Our CRISPR/Cas9 system effectively inhibited proliferation of human papillomavirus (HPV) 16-positive cervical cancer cells and induced apoptosis by inactivating the HR-HPV16E6/E7 oncogene. Based on this system, we prepared a long-circulating pH-sensitive cationic nano-liposome complex with a high cell targeting and gene knockout rate. Intratumoral injection of cationic liposomes targeted to splicing HPV16 E6/E7 in nude mice significantly inhibited tumor growth without significant toxicity . Liposomes that targeted HPV16 E6/E7 splicing were established as a basis for treatment of HPV16-positive cervical cancer drug candidates. Our study demonstrates that this liposome offers an efficient delivery system for nonviral gene editing, adding to the armamentarium of gene editing tools to advance safe and effective precision medicine-based cancer therapeutics.
Download full-text PDF |
Source |
---|---|
http://dx.doi.org/10.1089/hum.2019.312 | DOI Listing |
Front Microbiol
December 2024
National Key Laboratory of Intelligent Tracking and Forecasting for Infectious Disease, National Institute for Communicable Disease Control and Prevention, Chinese Center for Disease Control and Prevention, Beijing, China.
Background: strains are important opportunistic pathogens with great potential applications in industry and agriculture. Their significant genetic and phenotypic diversity has led to several changes in their taxonomic localization and was prone to inaccurate species classification based on traditional identification methods.
Methods: All 2,615 genomes of the genus were obtained from the NCBI genome database.
Front Genome Ed
December 2024
Department of Medical Cell Biology, Uppsala University, Uppsala, Sweden.
CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged as a potent gene-editing tool after the successful engineering of its synthetic guide RNA (sgRNA) toward the targeting of specific DNA sequences with high accuracy. Besides its DNA editing ability, further-developed Cas variants can also edit the epigenome, rendering the CRISPR-Cas system a versatile tool for genome and epigenome manipulation and a pioneering force in precision medicine. This review explores the latest advancements in CRISPR-Cas technology and its therapeutic and biomedical applications, highlighting its transformative impact on precision medicine.
View Article and Find Full Text PDFFront Pediatr
December 2024
Maternity and Child Health Care of Guangxi Zhuang Autonomous Region, Nanning, China.
Objective: to evaluate the research status and development hotspots of fetal α-thalassemia by quantitatively analyzing the diagnostic status, key areas, related management measures and prospects of the disease by bibliometrics.
Methods: The global literature on fetal α-thalassemia and severe α-thalassemia from 2009-2023 in the Web of Science Core Collection (WOSCC) was visually analyzed by VOSviewer and CiteSpace.
Results: (1) The examination of the quantity of publications concerning fetal α-thalassemia indicates a rising tendency prior to 2018, followed by a decrease after 2018.
Biotechnol Lett
December 2024
Jiangsu Key Laboratory for Pathogens and Ecosystems, College of Life Sciences, Nanjing Normal University, No.1 Wenyuan Rd., Xixia District, Nanjing, 210023, Jiangsu, People's Republic of China.
Recombineering (recombination-mediated genetic engineering) is a powerful strategy for bacterial genomic DNA and plasmid DNA modifications. CoS-MAGE improved over MAGE (multiplex automated genome engineering) by co-electroporation of an antibiotic resistance repair oligo along with the oligos for modification of the Escherichia coli chromosome. After several cycles of recombineering, the sub-population of mutants were selected among the antibiotic resistant colonies.
View Article and Find Full Text PDFJ Genet Genomics
December 2024
MOE Key Laboratory of Gene Function and Regulation, State Key Laboratory of Biocontrol, School of Life Sciences, Sun Yat-sen University, Guangzhou, Guangdong 510275, China; Key Laboratory of Reproductive Medicine of Guangdong Province, the First Affiliated Hospital and School of Life Sciences, Sun Yat-sen University, Guangzhou, Guangdong 510275, China. Electronic address:
Mutations in the Rhodopsin (RHO) gene are the main cause of autosomal dominant retinitis pigmentosa (adRP), 84% of which are pathogenic gain-of-function point mutations. Treatment strategies for adRP typically involve silencing or ablating the pathogenic allele, while normal RHO protein replacement has no meaningful therapeutic benefit. Here, we present an adenine base editor (ABE)-mediated therapeutic approach for adRP caused by RHO point mutations in vivo.
View Article and Find Full Text PDFEnter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!