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Molecular engineering of dendrimer nanovectors for siRNA delivery and gene silencing

Yu Cao, Xiaoxuan Liu, Ling Peng

Frontiers of Chemical Science and Engineering 2017, Volume 11, Issue 4,   Pages 663-675 doi: 10.1007/s11705-017-1623-5

Abstract: Small interfering RNA (siRNA) therapeutics hold great promise to treat a variety of diseases, as long as they can be delivered safely and effectively into cells. Dendrimers are appealing vectors for siRNA delivery by virtue of their well-defined molecular architecture and multivalent cooperativity. However, the clinical translation of RNA therapeutics mediated by dendrimer delivery is hampered by the lack of dendrimers that are of high quality to meet good manufacturing practice standard. In this context, we have developed small amphiphilic dendrimers that self-assemble into supramolecular structures, which mimic high-generation dendrimers synthesized with covalent construction, yet are easy to produce in large amount and superior quality. Indeed, the concept of supramolecular dendrimers has proved to be very promising, and has opened up a new avenue for dendrimer-mediated siRNA delivery. A series of self-assembling supramolecular dendrimers have consequently been established, some of them out-performing the currently available nonviral vectors in delivering siRNA to various cell types and , including human primary cells and stem cells. This short review presents a brief introduction to RNAi therapeutics, the obstacles to their delivery and the advantages of dendrimer delivery vectors as well as our bio-inspired structurally flexible dendrimers for siRNA delivery. We then highlight our efforts in creating self-assembling amphiphilic dendrimers to construct supramolecular dendrimer nanosystems for effective siRNA delivery as well as the related structural alterations to enhance delivery efficiency. The advent of self-assembling supramolecular dendrimer nanovectors holds great promise and heralds a new era of dendrimer-mediated delivery of RNA therapeutics in biomedical applications.

Keywords： gene therapy     RNAi therapeutics     dendrimer     nanovectors     gene silencing    

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Profiling influences of gene overexpression on heterologous resveratrol production in

Duo Liu,Bingzhi Li,Hong Liu,Xuejiao Guo,Yingjin Yuan

Frontiers of Chemical Science and Engineering 2017, Volume 11, Issue 1,   Pages 117-125 doi: 10.1007/s11705-016-1601-3

Abstract: Metabolic engineering of heterologous resveratrol production in faces challenges as the precursor L-tyrosineWe overexpressed the main gene targets in the upstream pathways to investigate their influences on theSingle-gene overexpression and DNA assembly-directed multigene overexpression affect the production ofFinally, the collaboration of selected gene targets leads to an optimal resveratrol production of 66.14The newly discovered gene targets expressing phosphoribosylanthranilate isomerase, expressing 3-deoxy-D-arabino-heptulosonate

Keywords： resveratrol     aromatic amino acid     DNA assembly     metabolic engineering     gene overexpression    

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Solution to Present Gene Engineering with ECIWO Theory——Techniques of Acquiring Polygene and Intensifying

Wang Ruiku

Strategic Study of CAE 2006, Volume 8, Issue 5,   Pages 25-28

Abstract: ECIWO Theory can solve some problems in gene transfer, which opens a new path to biological genetic operationThe paper discussed the theoretical basis and technical principle of ECIWO gene engineering, which is

Keywords： ECIWO     gene engineering     solution     acquiring polygene     intensifying anticipant characters    

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Advances in genetic engineering of domestic animals

Shaohua WANG,Kun ZHANG,Yunping DAI

Frontiers of Agricultural Science and Engineering 2016, Volume 3, Issue 1,   Pages 1-10 doi: 10.15302/J-FASE-2016085

Abstract: Thus, genetic engineering of domestic animals presents a critical and valuable tool to address theseOverall, genetic engineering has evolved through three stages in history: transgenesis, gene targeting, and gene editing.Since the birth of the first transgenic pig, genetic engineering in livestock has been advancing slowlyHowever, the low efficiency of gene targeting events in somatic cells prohibits its wide use in agricultural

Keywords： CRISPR     TALEN     ZFN     gene editing     livestock     pig     cattle    

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The Industry of Genetic Engineering

Shi Yuanchun

Strategic Study of CAE 2000, Volume 2, Issue 7,   Pages 34-38

Abstract:

This article explains that modern biotechnology not only has a powerful spreading, driving force and economic value, but it also reserves a great potential to relax the global pressures from population, food, natural resources and environment. None of ohter technologies have such a power, or can replace biotechnology. The article also discusses the main contents of biotechnology and its new trends of industrialization. The author holds GMO food must be put in order by both science and relevant regulations, after reviewed the background and new trends of present GMO debate. The current debate has focused on the public acceptability, trade benefit and relevant policy on GMO food. GMO food will only be interrupted for the time being, and will have another development tide under the supervision of relevant laws and regulation. Finally, the paper proposes suggestions for the countermeasure and attitude that China should hold during the development and industrialization of modern biotechnology.

Keywords： genetic engineering     industry     gene modified organisms (GMO)     countermeasure    

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Erratum to “Targeted Genotyping of a Whole-Gene Repertoire by an Ultrahigh-Multiplex and Flexible HD-MarkerApproach” [Engineering 13 (2022) 186–196] Erratum

Pingping Liu,Jia Lv,Cen Ma,Tianqi Zhang,Xiaowen Huang,Zhihui Yang,Lingling Zhang,Jingjie Hu,Shi Wang,Zhenmin Bao,

Engineering 2022, Volume 18, Issue 11,   Pages 259-259 doi: 10.1016/j.eng.2022.09.002

Keywords： online     THE AUTHORS.Published     repertoire     behalf     Engineering     September     genotyping     Academy     HD-marker approach    

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Study on Family Collection, Gene Mapping, Gene Identification and Gene Function of Human Genetic Diseases

Xia Jiahui

Strategic Study of CAE 2000, Volume 2, Issue 11,   Pages 1-11

Abstract:

In this paper, the study on family collection, gene mapping, gene identification and gene functiont (1; 3) (q44; p11) associated with nasopharyngeal cancer was found in 1975 at first, and human TDF geneIn 1998, GJB3, a human genetic neurological deafness gene, was identified using a novel strategy of “Gene Family-Candidate Disease Gene Cloning”，and the paper was published in Nature Geneticswas also cloned from gene function study.

Keywords： genetic disease     gene mapping and cloning     gene familiy-candidate disease gene cloning     genome wide scan     gene function study    

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The antibiotic resistome: gene flow in environments, animals and human beings

Yongfei Hu, George F. Gao, Baoli Zhu

Frontiers of Medicine 2017, Volume 11, Issue 2,   Pages 161-168 doi: 10.1007/s11684-017-0531-x

Abstract: We propose that the resistance gene flow in nature is “from the natural environments” andand animals, as intermediate recipients and disseminators, contribute greatly to such a resistance gene

Keywords： antibiotic resistance     resistome     microbiome     gene flow    

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Reflections on the system of evaluation of gene-edited livestock

Ziyao FAN, Tianwen WU, Kui WU, Yulian MU, Kui LI

Frontiers of Agricultural Science and Engineering 2020, Volume 7, Issue 2,   Pages 211-217 doi: 10.15302/J-FASE-2019303

Abstract: functions regarding economic traits, and new materials for livestock breeding have been obtained through geneEvaluation is the most important component of the regulation of gene-edited livestock and is a crucialguarantee of public safety before the marketing of gene-edited animal products.Here, the system of evaluation of gene-edited livestock is discussed in terms of public safety and production

Keywords： evaluation     gene editing     livestock     performance     safety    

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Distinct gene expression pattern of mutations coordinated by target repression and promoter hypermethylation

Frontiers of Medicine 2022, Volume 16, Issue 4,   Pages 627-636 doi: 10.1007/s11684-020-0815-4

Abstract: Then, based on RNA-seq data from The Cancer Genome Atlas AML cohort, we developed a 300-gene signatureThis study revealed the distinct gene expression pattern of RUNX1 mutations and the underlying

Keywords： RUNX1     gene mutation     acute myeloid leukemia     transcriptional repression     DNA methylation    

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Construction of lentiviral vector carrying Rab9 gene and its expression in mouse brain

Youguo HAO, Min ZHANG, Jinzhi XU, Bitao BU, Jiajun WEI

Frontiers of Medicine 2009, Volume 3, Issue 2,   Pages 141-147 doi: 10.1007/s11684-009-0041-6

Abstract: To explore the possibility of Rab9-related gene therapy for neurodegenerative diseases, we packed LentivirusWestern blotting revealed that the Rab9 gene expression in BALB/c mice brain was up-regulated significantlyLentivirus encoding Rab9 can increase the expression of Rab9 gene effectively, which might offer a novel

Keywords： Rab9     lentivirus     gene therapy     gene transfer    

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Gene delivery into isolated

Nan Zheng, Ziyuan Song, Yang Liu, Lichen Yin, Jianjun Cheng

Frontiers of Chemical Science and Engineering 2017, Volume 11, Issue 4,   Pages 521-528 doi: 10.1007/s11705-017-1612-8

Abstract: The application of gene delivery materials has been mainly focused on mammalian cells while rarely extendedto plant engineering.This study therefore widens the utilities of helical polypeptide as a unique category of gene deliverymaterials, and may find their promising applications toward plant gene delivery.

Keywords： α-helical polypeptide     plant gene delivery     protoplast     intact leaves     transfection    

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Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

Chuanfeng Wu, Cynthia E. Dunbar

Frontiers of Medicine 2011, Volume 5, Issue 4,   Pages 356-371 doi: 10.1007/s11684-011-0159-1

Abstract: Virus-based vectors are widely used in hematopoietic stem cell (HSC) gene therapy, and have the abilityTo date, HSC gene therapy has been successfully employed in the clinic for improving clinical outcomesHowever, adverse events were observed during some of these HSC gene therapy clinical trials, linked tomechanisms of insertional mutagenesis in hematopoietic stem and progenitor cells due to integrating gene, and introduce newly-developed approaches for minimizing genotoxicity as a way to further move HSC gene

Keywords： gene therapy     hematopoietic stem cells     insertional mutagenesis     genotoxicity     induced pluripotent stem cell    

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Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B

Ru Zhang,Qiang Wang,Lin Zhang,Saijuan Chen

Frontiers of Medicine 2015, Volume 9, Issue 1,   Pages 90-99 doi: 10.1007/s11684-015-0390-2

Abstract:

Gene therapy provides a potential cure for hemophilia B, and significant progress has been achievedin liver-directed gene transfer mediated by adeno-associated viral vectors.vectors and hFIX expression cassette may positively contribute to the ultimate success of hemophilia B geneIn addition, the regulatory elements that control the FIX gene expression in these cassettesreconstructs of hFIX with AAV vector may potentially contribute to the creation of an efficacious gene

Keywords： factor IX     hemophilia B     liver-specific regulatory elements     hydrodynamic gene transfer    

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Identification of cancer gene fusions based on advanced analysis of the human genome or transcriptome

Lu Wang

Frontiers of Medicine 2013, Volume 7, Issue 3,   Pages 280-289 doi: 10.1007/s11684-013-0265-3

Abstract:

Many gene fusions have been recognized as important diagnostic and/or prognostic markers in humanIn recent years, novel gene fusions have been identified in cases without prior knowledge of the geneticThis review focused on advanced genome-wide screening approaches in fusion gene identification, suchas microarray-based approaches, next-generation sequencing, and NanoString nCounter gene expression systemThe fundamental rationale and strategy for fusion gene identification using each biotech platform are

Keywords： gene fusion     cancer     microarray     next-generation sequencing     NanoString nCounter system    

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Title Author Date Type Operation

Molecular engineering of dendrimer nanovectors for siRNA delivery and gene silencing

Yu Cao, Xiaoxuan Liu, Ling Peng

Journal Article

Profiling influences of gene overexpression on heterologous resveratrol production in

Duo Liu,Bingzhi Li,Hong Liu,Xuejiao Guo,Yingjin Yuan

Journal Article

Solution to Present Gene Engineering with ECIWO Theory——Techniques of Acquiring Polygene and Intensifying

Wang Ruiku

Journal Article

Advances in genetic engineering of domestic animals

Shaohua WANG,Kun ZHANG,Yunping DAI

Journal Article

The Industry of Genetic Engineering

Shi Yuanchun

Journal Article

Erratum to “Targeted Genotyping of a Whole-Gene Repertoire by an Ultrahigh-Multiplex and Flexible HD-MarkerApproach” [Engineering 13 (2022) 186–196]

Pingping Liu,Jia Lv,Cen Ma,Tianqi Zhang,Xiaowen Huang,Zhihui Yang,Lingling Zhang,Jingjie Hu,Shi Wang,Zhenmin Bao,

Journal Article

Study on Family Collection, Gene Mapping, Gene Identification and Gene Function of Human Genetic Diseases

Xia Jiahui

Journal Article

The antibiotic resistome: gene flow in environments, animals and human beings

Yongfei Hu, George F. Gao, Baoli Zhu

Journal Article

Reflections on the system of evaluation of gene-edited livestock

Ziyao FAN, Tianwen WU, Kui WU, Yulian MU, Kui LI

Journal Article

Distinct gene expression pattern of mutations coordinated by target repression and promoter hypermethylation

Journal Article

Construction of lentiviral vector carrying Rab9 gene and its expression in mouse brain

Youguo HAO, Min ZHANG, Jinzhi XU, Bitao BU, Jiajun WEI

Journal Article

Gene delivery into isolated

Nan Zheng, Ziyuan Song, Yang Liu, Lichen Yin, Jianjun Cheng

Journal Article

Stem cell gene therapy: the risks of insertional mutagenesis and approaches to minimize genotoxicity

Chuanfeng Wu, Cynthia E. Dunbar

Journal Article

Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B

Ru Zhang,Qiang Wang,Lin Zhang,Saijuan Chen

Journal Article

Identification of cancer gene fusions based on advanced analysis of the human genome or transcriptome

Lu Wang

Journal Article