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Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine

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Genes & Diseases 2024年第1期11卷 268-282页

作者： Mohadeseh Khoshandam Hossein Soltaninejad Marziyeh Mousazadeh Amir Ali Hamidieh Saman Hosseinkhani Department of Reproductive Biology Academic Center for EducationCultureand Research(ACECR)Qom BranchQom ***Iran National Institute of Genetic Engineering and Biotechnology(NIGEB) Tehran 14155-6463Iran Faculty of Interdisciplinary Science and Technology Tarbiat Modares UniversityTehran 14117-13116Iran Pediatric Cell and Gene Therapy research center GeneCell&Tissue Research InstituteTehran UniversityofMedical SciencesTehran14155-6559Iran Department of Nanobiotechnology Faculty of Biological SciencesTarbiat Modares UniversityTehran 14117-13116Iran Department of Biochemistry Faculty of Biological SciencesTarbiat Modares UniversityTehran 14117-13116Iran

CRISPR/Cas9 is an effective gene editing tool with broad applications for the pre-vention or treatment of numerous *** depends on CRiSPR(clustered regularly inter-spaced short palindromic repeats)as a bacterial immune system and plays as a gene editing *** to the higher specificity and efficiency of CRISPR/Cas9 compared to other editing ap-proaches,it has been broadly investigated to treat numerous hereditary and acquired ill-nesses,including cancers,hemolytic diseases,immunodeficiency disorders,cardiovascular diseases,visual maladies,neurodegenerative conditions,and a few X-linked ***/Cas9 system has been used to treat cancers through a variety of approaches,with sta-ble gene editing ***,the applications and clinical trials of CRisPR/Cas9 in various illnesses are *** to its high precision and efficiency,CRISPR/Cas9 strategies may treat gene-related illnesses by deleting,inserting,modifying,or blocking the expression of specific *** most challenging barrier to the in vivo use of CRISPR/Cas9 like off-target effects will be *** use of transfection vehicles for CRISPR/Cas9,including viral vectors(such as an Adeno-associated virus(AAV),and the development of non-viral vectors is also considered.

关键词： Clinical trials CRISPR/Cas9 Gene therapy Non-viral vectors Viral vectors

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Two different autorefractors for vision screening in children and adolescents

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International Journal of Ophthalmology(English edition) 2024年第2期17卷 331-338页

作者： Xiao-Jun Chen Lin-Jie Liu Bing Sun Dan-Dan Jiang Shu-Shu Zuo Yan-Hui Wang Chun-Hua Zhang Yan-Yan Chen School of Optometry and Ophthalmology Wenzhou Medical UniversityWenzhou 325027Zhejiang ProvinceChina National Clinical research center for Ocular Diseases Eye HospitalWenzhou Medical UniversityWenzhou 325027Zhejiang ProvinceChina

AIM:To compare the consistency of two autorefractors(Tianle RM-9000 and Topcon KR-800)for school-age myopia children,and to provide a basis for largescale data analysis and ***:The refractive error in 909 subjects(age 4–18y)were measured using both autorefractors without *** data were analyzed using Fourier decomposition and the correlation coefficients,intraclass correlation coefficients(ICC),and Bland-Altman limits of agreement(LoA)for each parameter were ***:There was a strong correlation between the spherical equivalent(SE),sphere diopter(DS),and cylinder diopter(DC)readings of the Tianle RM-9000 and those of the Topcon KR-800,with correlation coefficient values of 0.98,0.98 and 0.83 and ICC values of 0.99,0.99 and 0.93,***,the correlation coefficients and ICC values of J0 and J45 were unreliable(R=-0.004,-0.034;both ICCvectors are unreliable and should not be used to assess astigmatism.

关键词： refractive error autorefractors school-age myopia children

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Reanalysis of energy band structure in the type-II quantum wells

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Chinese Physics B 2024年第6期33卷 75-78页

作者：李欣欣邓震江洋杜春花贾海强王文新陈弘 Key Laboratory for Renewable Energy Beijing Key Laboratory for New Energy Materials and DevicesBeijing National Laboratory for Condensed Matter PhysicsInstitute of PhysicsChinese Academy of SciencesBeijing 100190China center of Materials and Optoelectronics Engineering University of Chinese Academy of SciencesBeijing 100049China The Yangtze River Delta Physics research center Liyang 213000China Songshan Lake Materials Laboratory Dongguan 523808China

Band structure analysis holds significant importance for understanding the optoelectronic characteristics of semiconductor structures and exploring their potential applications in practice. For quantum well structures, the energy of carriers in the well splits into discrete energy levels due to the confinement of barriers in the growth direction. However, the discrete energy levels obtained at a fixed wave vector cannot accurately reflect the actual energy band structure. In this work, the band structure of the type-II quantum wells is reanalyzed. When the wave vectors of the entire Brillouin region(corresponding to the growth direction) are taken into account, the quantized energy levels of the carriers in the well are replaced by subbands with certain energy distributions. This new understanding of the energy bands of low-dimensional structures not only helps us to have a deeper cognition of the structure, but also may overturn many viewpoints in traditional band theories and serve as supplementary to the band theory of low-dimensional systems.

关键词： energy band structure type-II quantum wells low-dimensional semiconductors

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Comprehensive Relation Modelling for Image Paragraph Generation

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Machine Intelligence research 2024年第2期21卷 369-382页

作者： Xianglu Zhu Zhang Zhang Wei Wang Zilei Wang Automation Department University of Science and Technology of ChinaHefei 230027China center for research on Intelligent Perception and Computing National Laboratory of Pattern RecognitionInstitute of AutomationChinese Academy of SciencesBeijing 100190China University of Chinese Academy of Sciences Beijing 100864China

Image paragraph generation aims to generate a long description composed of multiple sentences,which is different from traditional image captioning containing only one *** of previous methods are dedicated to extracting rich features from image regions,and ignore modelling the visual *** this paper,we propose a novel method to generate a paragraph by modelling visual relationships ***,we parse an image into a scene graph,where each node represents a specific object and each edge denotes the relationship between two ***,we enrich the object features by implicitly encoding visual relationships through a graph convolutional network(GCN).We further explore high-order relations between different relation features using another graph convolutional *** addition,we obtain the linguistic features by projecting the predicted object labels and their relationships into a semantic embedding *** these features,we present an attention-based topic generation network to select relevant features and produce a set of topic vectors,which are then utilized to generate multiple *** evaluate the proposed method on the Stanford image-paragraph dataset which is currently the only available dataset for image paragraph generation,and our method achieves competitive performance in comparison with other state-of-the-art(SOTA)methods.

关键词： Image paragraph generation visual relationship scene graph graph convolutional network(GCN) long short-term memory

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Engineered AAV13 variants with enhanced transduction and confined spread

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Zoological research 2024年第4期45卷 781-790页

作者： Neng-Song Luo Yu-Xiang Cai Zeng-Peng Han Xiao-Kai Sui Wen-Jia Yuan Zi-Lian Zhang Hao-Dong Guo Jie Wang Kun-Zhang Lin Fu-Qiang Xu Wuhan National Laboratory for Optoelectronics Huazhong University of Science and TechnologyWuhanHubei 430074China Shenzhen Key Laboratory of Viral vectors for Biomedicine Shenzhen-Hong Kong Institute of Brain ScienceShenzhen Institute of Advanced TechnologyChinese Academy of SciencesShenzhenGuangdong 518055China Key Laboratory of Magnetic Resonance in Biological Systems State Key Laboratory of Magnetic Resonance and Atomic and Molecular PhysicsNational Center for Magnetic Resonance in WuhanInnovation Academy for Precision Measurement Science and TechnologyChinese Academy of SciencesWuhanHubei 430071China Key Laboratory of Quality Control Technology for Virus-Based Therapeutics Guangdong Provincial Medical Products AdministrationNMPA Key Laboratory for Research and Evaluation of Viral Vector Technology in Cell and Gene Therapy Medicinal ProductsBrain Cognition and Brain Disease InstituteShenzhen Institute of Advanced TechnologyChinese Academy of SciencesShenzhenGuangdong 518055China University of Chinese Academy of Sciences Beijing 100049China Zhongnan Hospital Wuhan UniversityWuhanHubei 430072China Brain Case Biotechnology Co. Ltd.ShenzhenGuangdong 518107China College of Life Sciences Wuhan UniversityWuhanHubei 430072China center for Excellence in Brain Science and Intelligence Technology Chinese Academy of SciencesShanghai 200031China

Precise targeting of specific regions within the central nervous system(CNS)is crucial for both scientific research and gene therapy in the context of brain ***-associated virus 13(AAV13)is known for its restricted diffusion range within the CNS,making it an ideal choice for precise labeling and administration within small brain ***,AAV13 mediates relatively low expression of target ***,we introduced specifically engineered modifications to the AAV13 capsid protein to enhance its transduction *** first constructed AAV13-YF by mutating tyrosine to phenylalanine on the surface of the AAV13 *** then inserted the 7m8 peptide,known to enhance cell transduction,into positions 587/588 and 585/586 of the AAV13 capsid,resulting in two distinct variants named AAV13-587-7m8 and AAV13-585-7m8,*** found that AAV13-YF exhibited superior in vitro infectivity in HEK293T cells compared to AAV13,while AAV13-587-7m8 and AAV13-585-7m8 showed enhanced CNS infection capabilities in C57BL/6 mice,with AAV13-587-7m8 infection retaining a limited spread *** modified AAV13 variants hold promising potential for applications in gene therapy and neuroscience research.

关键词： Adeno-associated virus 13 AAV13-YF AAV13-587-7m8 AAV13-585-7m8 transduction efficiency

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Mitigation on self-discharge behaviors via morphological control of hierarchical Ni-sulfides/Ni-oxides electrodes for long-life-supercapacitors

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Journal of Materials Science & Technology 2022年第18期113卷 217-228页

作者： Dhananjay Mishra Niraj Kumar Ajit Kumar SeungGi Seo SungHun Jin Department of Electronic Engineering Incheon National UniversityIncheon 406-772 and also with Convergence Research Center for Insect VectorsIncheon National UniversityRepublic of Korea

To cope up with the sustainable energy storage goals for supercapacitors(SCs),the self-discharge in SC electrodes is a significant hurdle,and thereby,nickel sulfide(NS)with high conductivity is adopted as a test vehicle for understanding the morphological evolution effects for long-life ***,honeycomb-like NS is hierarchically formed over hydrothermally grown nickel oxide(NO)via successive ionic layer adsorption reaction(SILAR)*** heterostructure shows a fivefold improvement in specific capacitance from 348 F g^(−1) to 2077 F g^(−1)at 1 mV s^(−1) over bare ***,the remarkable upliftment of capacitance retention is achieved from 60.7%to 92.3%even after 3000 cycles via morphological control of NS/NO hetero-structure with the help of highly conductive *** importantly,the self-discharge behaviors and synergistic role of leakage current associated with morphological evolution via NS overcoating are studied in *** particular,the self-discharge mitigation from 45%(NO)to 35%(NS20/NO)due to the NS/NO heterostructure and the behind mechanism are ascribed to the activated-controlled Faradaic reaction coupled with a charge *** study emphasizes the potential importance of composite heterostructure by tuning the electrical conductivity and morphological adjustment NO via consecutive overcoating of NS through SILAR as a novel *** enhances charge storage,redox kinetics,and the mitigation of self-discharge properties of the active electrode *** practical validation on sustainable energy storage,NS20/NO supercapacitors illuminate the LED for 35%longer than NO after one-time charging,potentially beneficial for the next generation SCs.

关键词： Morphological evolution Self-discharge SILAR overcoating Supercapacitors Heterostructure

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Secure Transmission of Compressed Medical Image Sequences on Communication Networks Using Motion Vector Watermarking

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Computers, Materials & Continua 2024年第3期78卷 3283-3301页

作者： Rafi Ullah Mohd Hilmi bin Hasan Sultan Daud Khan Mussadiq Abdul Rahim Computer and Information Sciences Department Universiti Teknologi PETRONASBandar Seri Iskandar Perak32610Malaysia Department of Computer Science National University of TechnologyIslamabad46000Pakistan Interdisciplinary research center for Intelligent Secure Systems King Fahd University of Petroleum and MineralsDammam31261Saudi Arabia

Medical imaging plays a key role within modern hospital management systems for diagnostic *** methodologies are extensively employed to mitigate storage demands and enhance transmission speed,all while upholding image ***,an increasing number of hospitals are embracing cloud computing for patient data storage,necessitating meticulous scrutiny of server security and privacy ***,considering the widespread availability of multimedia tools,the preservation of digital data integrity surpasses the significance of compression *** response to this concern,we propose a secure storage and transmission solution for compressed medical image sequences,such as ultrasound images,utilizing a motion vector watermarking *** watermark is generated employing an error-correcting code known as Bose-Chaudhuri-Hocquenghem(BCH)and is subsequently embedded into the compressed sequence via block-based motion *** the process of watermark embedding,motion vectors are selected based on their magnitude and phase *** embedding watermarks,no specific spatial area,such as a region of interest(ROI),is used in the *** embedding of watermark bits is dependent on motion *** reversible watermarking allows the restoration of the original image sequences,we use the irreversible watermarking *** reason for this is that the use of reversible watermarks may impede the claims of ownership and legal *** restoration of original data or images may call into question ownership or other legal *** peak signal-to-noise ratio(PSNR)and structural similarity index(SSIM)serve as metrics for evaluating the watermarked image *** all images,the PSNR value exceeds 46 dB,and the SSIM value exceeds *** results substantiate the efficacy of the proposed technique in preserving data integrity.

关键词： Block matching algorithm(BMA) compression full-search algorithm motion vectors ultrasound image sequence watermarking

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Biotin-modified Galactosylated Chitosan-gene Carrier in Hepatoma Cells Targeting Delivery

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Journal of Wuhan University of Technology(Materials Science) 2024年第2期39卷 522-531页

作者：程明荣张锋李清王华 Department of General Surgery Nanxiang Branch of Ruijin HospitalShanghai 201802China Minhang District Pujin Community Health Service center Shanghai 201112China Hepatobiliary Surgery center Tongji Hospital Affiliated to Tongji UniversityShanghai 200065China

Our previous studies have successfully grafted biotin and galactose onto chitosan(CS)and synthesized biotin modified galactosylated chitosan(Bio-GC).The optimum N/P ratio of Bio-GC and plasmid DNA was 3:*** this N/P ratio,the transfection efficiency in the hepatoma cells was the highest with a slow release ***-GC nanomaterials exhibit the protective effect of preventing the gene from nuclease degradation,and can target the transfection into hepatoma cells by combination with galactose and biotin *** transfection rate was inhibited by the competition of galactose and ***-GC nanomaterials were imported into cells’cytoplasm by their receptors,followed by the imported exogenous gene transfected into the ***-GC nanomaterials can also cause inhibitory activity in the hepatoma cells in the model of orthotopic liver transplantation in mice,by carrying the gene through the blood to the hepatoma *** together,bio-GC nanomaterials act as gene vectors with the activity of protecting the gene from DNase degradation,improving the rate of transfection in hepatoma cells,and transporting the gene into the cytoplasm in vitro and in ***,they are efficient hepatoma-targeting gene carriers.

关键词： gene vector hepatocellular carcinoma nanoparticles sustained release gene therapy

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Species-level Microbiota of Biting Midges and Ticks from Poyang Lake

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Biomedical and Environmental Sciences 2024年第3期37卷 266-277,I0001-I0003页

作者： GONG Jian WANG Fei Fei LIU Yang Qing PU Ji DONG Ling Zhi ZHANG Si Hui HUANG Zhen Zhou HUANG Yu Yuan LI Ya Ben YANG Cai Xin TAO Yuan Meihui ZHAO Li Jun JIN Dong LIU Li Yun YANG Jing LU Shan Department of Epidemiology School of Public HealthShanxi Medical UniversityTaiyuan 030001ShanxiChina National Key Laboratory of Intelligent Tracking and Forecasting for Infectious Diseases National Institute for Communicable Disease Control and PreventionChinese Center for Disease Control and PreventionBeijing 102206China Jiangxi Provincial Key Laboratory of Animal-Origin and Vector-Borne Diseases Nanchang Center for Disease Control and PreventionNanchang 330038JiangxiChina research Units of Discovery of Unknown Bacteria and Function Chinese Academy of Medical SciencesBeijing 100730China

Objective The purpose of this study was to investigate the bacterial communities of biting midges and ticks collected from three sites in the Poyang Lake area,namely,Qunlu Practice Base,Peach Blossom Garden,and Huangtong Animal Husbandry,and whether vectors carry any bacterial pathogens that may cause diseases to humans,to provide scientific basis for prospective pathogen discovery and disease prevention and *** Using a metataxonomics approach in concert with full-length 16S rRNA gene sequencing and operational phylogenetic unit(OPU)analysis,we characterized the species-level microbial community structure of two important vector species,biting midges and ticks,including 33 arthropod samples comprising 3,885 individuals,collected around Poyang *** A total of 662 OPUs were classified in biting midges,including 195 known species and 373 potentially new species,and 618 OPUs were classified in ticks,including 217 known species and 326 potentially new ***,OPUs with potentially pathogenicity were detected in both arthropod vectors,with 66 known species of biting midges reported to carry potential pathogens,including Asaia lannensis and Rickettsia bellii,compared to 50 in ticks,such as Acinetobacter lwoffii and Staphylococcus *** found that Proteobacteria was the most dominant group in both midges and ***,the outcomes demonstrated that the microbiota of midges and ticks tend to be governed by a few highly abundant *** sp7 was predominant in biting midges,while Coxiella sp1 was enriched in ***,Coxiella spp.,which may be essential for the survival of Haemaphysalis longicornis Neumann,were detected in all tick *** identification of dominant species and pathogens of biting midges and ticks in this study serves to broaden our knowledge associated to microbes of arthropod *** Biting midges and ticks carry large numbers of known and potentially novel bacteria,and carry a wide

关键词： Biting midge Tick Microbiota Pantoea Coxiella

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Genomics-driven derivatization of the bioactive fungal sesterterpenoid variecolin:Creation of an unnatural analogue with improved anticancer properties

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Acta Pharmaceutica Sinica B 2024年第1期14卷 421-432页

作者： Dexiu Yan Jemma Arakelyan Teng Wan Ritvik Raina Tsz Ki Chan Dohyun Ahn Vladimir Kushnarev Tsz Kiu Cheung Ho Ching Chan Inseo Choi Pui Yi Ho Feijun Hu Yujeong Kim Hill Lam Lau Ying Lo Law Chi Seng Leung Chun Yin Tong Kai Kap Wong Wing Lam Yim Nikolay S.Karnaukhov Richard Y.C.Kong Maria V.Babak Yudai Matsuda Department of Chemistry City University of Hong KongHong Kong SARChina iGEM Team“VarieCure” City University of Hong KongHong Kong SARChina Department of Biomedical Engineering City University of Hong KongHong Kong SARChina Moscow Clinical research center Named After A.S.Loginov Moscow 111123Russian Federation

A biosynthetic gene cluster for the bioactive fungal sesterterpenoids variecolin(1)and variecolactone(2)was identified in Aspergillus aculeatus ATCC *** production of 1 and 2 was achieved in Aspergillus oryzae by expressing the sesterterpene synthase VrcA and the cytochrome P450 ***,the replacement of VrcB with homologous P450s from other fungal terpenoid pathways yielded three new variecolin analogues(5-7).Analysis of the compounds’anticancer activity in vitro and in vivo revealed that although 5 and 1 had comparable activities,5 was associated with significantly reduced toxic side effects in cancer-bearing mice,indicating its potentially broader therapeutic *** study describes the first tests of variecolin and its analogues in animals and demonstrates the utility of synthetic biology for creating molecules with improved biological activities.

关键词： Animal studies Anticancer properties Biosynthesis Natural products Sesterterpenoids Synthetic biology Terpene synthases Variecolin

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