Therapeutic application of sequence-specific binding molecules for novel genome editing tools

2021 ◽  
pp. 100427
Author(s):  
Juki Nakao ◽  
Tsuyoshi Yamamoto ◽  
Asako Yamayoshi
Keyword(s):  
Genome Editing ◽  
Specific Binding ◽  
Therapeutic Application ◽  
Specific Binding Molecules

scholarly journals Dielectric Nanoparticles Coated upon Silver Hollow Nanosphere as an Integrated Design to Reinforce SERS Detection of Trace Ampicillin in Milk Solution

Coatings ◽  
2020 ◽  
Vol 10 (4) ◽  
pp. 390 ◽  
Author(s):  
Han Lee ◽  
Jia-Wei Yang ◽  
Jiunn-Der Liao ◽  
Jaya Sitjar ◽  
Bernard Haochih Liu ◽  
...  
Keyword(s):  
Active Sites ◽  
Specific Binding ◽  
Hot Spot ◽  
Ion Beam ◽  
Integrated Design ◽  
Small Diameter ◽  
Detection Sensitivity ◽  
Target Species ◽  
Specific Binding Molecules ◽  
Al2o3 Nps

Surface-enhanced Raman scattering (SERS) technique is competent to trace detection of target species, down to the single molecule level. The detection sensitivity is presumably degraded by the presence of non-specific binding molecules that occupy a SERS-active site (or hot spot) on the substrate surface. In this study, a silver hollow nano-sphere (Ag HNS) with cavity has been particularly designed, followed by depositing dielectric nanoparticles (Di NPs) upon Ag HNS. In the integrated nanostructures, Di NPs/Ag HNS were furthermore confirmed by cutting through the cross sections using the Focused Ion Beam (FIB) technique, which provides the Scanning Electron Microscope (SEM) with Energy-dispersive Spectroscope (EDS) mode for identifying the distribution of Di NPs upon Ag HNS. The results have indicated that Di NPs/Ag HNS exhibits small diameter of cavity, and among Di NPs in this study, Al2O3 with lower dielectric constant provides a much higher SERS enhancement factor (e.g., ~6.2 × 107). In this study, to detect trace amounts (e.g., 0.01 ppm) of Ampicillin in water or milk solution, Al2O3 NPs/Ag HNS was found to be more efficient and less influenced by non-specific binding molecules in milk. A substrate with integrated plasmonic and dielectric components was designed to increase the adsorption of target species and to repulse non-specific binding molecules from SERS-active sites.

Advances in therapeutic application of CRISPR-Cas9

2019 ◽  
Vol 19 (3) ◽  
pp. 164-174 ◽  
Author(s):  
Jinyu Sun ◽  
Jianchu Wang ◽  
Donghui Zheng ◽  
Xiaorong Hu
Keyword(s):  
Gene Therapy ◽  
Genome Editing ◽  
Malignant Tumor ◽  
Gene Editing ◽  
Genome Engineering ◽  
Therapeutic Application ◽  
Adaptive Immune ◽  
Efficient Gene

Abstract Clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) is one of the most versatile and efficient gene editing technologies, which is derived from adaptive immune strategies for bacteria and archaea. With the remarkable development of programmable nuclease-based genome engineering these years, CRISPR-Cas9 system has developed quickly in recent 5 years and has been widely applied in countless areas, including genome editing, gene function investigation and gene therapy both in vitro and in vivo. In this paper, we briefly introduce the mechanisms of CRISPR-Cas9 tool in genome editing. More importantly, we review the recent therapeutic application of CRISPR-Cas9 in various diseases, including hematologic diseases, infectious diseases and malignant tumor. Finally, we discuss the current challenges and consider thoughtfully what advances are required in order to further develop the therapeutic application of CRISPR-Cas9 in the future.

scholarly journals Gene editing in dermatology: Harnessing CRISPR for the treatment of cutaneous disease

F1000Research ◽  
2020 ◽  
Vol 9 ◽  
pp. 281
Author(s):  
Catherine Baker ◽  
Matthew S. Hayden
Keyword(s):  
Human Genome ◽  
Genome Editing ◽  
Gene Editing ◽  
Human Diseases ◽  
Therapeutic Application ◽  
Cutaneous Disease ◽  
Crispr System ◽  
Novel Approaches

The discovery of the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) system has revolutionized gene editing research. Through the repurposing of programmable RNA-guided CRISPR-associated (Cas) nucleases, CRISPR-based genome editing systems allow for the precise modification of specific sites in the human genome and inspire novel approaches for the study and treatment of inherited and acquired human diseases. Here, we review how CRISPR technologies have stimulated key advances in dermatologic research.  We discuss the role of CRISPR in genome editing for cutaneous disease and highlight studies on the use of CRISPR-Cas technologies for genodermatoses, cutaneous viruses and bacteria, and melanoma. Additionally, we examine key limitations of current CRISPR technologies, including the challenges these limitations pose for the widespread therapeutic application of CRISPR-based therapeutics.

Therapeutic application of genome editing in dyslipidemia

2021 ◽  
Vol Publish Ahead of Print ◽  
Author(s):  
Madelynn N. Whittaker ◽  
Kiran Musunuru
Keyword(s):  
Genome Editing ◽  
Therapeutic Application

scholarly journals Recent advances in genome editing of stem cells for drug discovery and therapeutic application

2020 ◽  
Vol 209 ◽  
pp. 107501 ◽  
Author(s):  
Jaesuk Lee ◽  
Delger Bayarsaikhan ◽  
Govigerel Bayarsaikhan ◽  
Jin-Soo Kim ◽  
Elizabeth Schwarzbach ◽  
...  
Keyword(s):  
Stem Cells ◽  
Drug Discovery ◽  
Genome Editing ◽  
Therapeutic Application ◽  
Recent Advances

scholarly journals Study of the binding mechanisms between palytoxin and its aptamer by docking and molecular simulation

2018 ◽  
Author(s):  
Bo Hu ◽  
Rong Zhou ◽  
Zhengang Li ◽  
Jiaxiang Qin ◽  
Shengqun Ouyang ◽  
...  
Keyword(s):  
Molecular Structure ◽  
Hydrogen Bonds ◽  
Specific Binding ◽  
Interaction Mechanism ◽  
Computational Results ◽  
Therapeutic Application ◽  
High Affinity ◽  
Ssdna Aptamer ◽  
Molecular Structure Analysis ◽  
Binding Mechanisms

This paper provides a feasible model for aptamer and its target in molecular structure analysis and interaction mechanism. In this study, modeling and dynamic simulation of ssDNA aptamer (P-18S2) and target (Palytoxin,PTX) were performed separately. Then, the combination mechanism of DNA and PTX were predicted, and docking results showed that PTX can combine steadily in the groove at the top of DNA model trough strong hydrogen-bonds and electrostatic interaction. Therefore, we have further truncated and optimized to P-18S2 by simulating, at the same time, we also confirmed the reliability of simulative results by experimenting. Combining with the experimental and computational results, we provide a more reasonable interpretation for the high affinity and specific binding of P-18S2 and PTX and offer the basis for aptamer development in molecular diagnostics and therapeutic application.

scholarly journals Affinity Capillary Electrophoresis for the Screening of Novel Antimicrobial Targets

2004 ◽  
Vol 9 (4) ◽  
pp. 303-308 ◽  
Author(s):  
L. Michelle Lewis ◽  
Linda J. Engle ◽  
William E. Pierceall ◽  
Dallas E. Hughes ◽  
Karen Joy Shaw
Keyword(s):  
Capillary Electrophoresis ◽  
Specific Binding ◽  
Resistance Mechanisms ◽  
Open Reading Frames ◽  
The Novel ◽  
Affinity Capillary Electrophoresis ◽  
Microbial Genomes ◽  
Efficient Detection ◽  
Novel Target ◽  
Specific Binding Molecules

The increasing number of multiantibiotic-resistant organisms, including methicillin-resistant Staphylococcus aureus (MRSA), requires the development of novel chemotherapies that are structurally distinct and exempt from current resistance mechanisms. Bioinformatics data mining of microbial genomes has revealed numerous previously unexploited essential open reading frames (ORFs) of unknown biochemical function. The potential of these proteins as screening targets is not readily apparent because most screening technologies rely on knowledge of biological function. To address this problem, the authors employed affinity capillary electrophoresis (ACE) to identify antimicrobial compounds that bound the novel target YihA. Screening a small-molecule library of 44,000 compounds initially identified 115 binders, of which 76% were confirmed. Furthermore, the ACE assay distinguished diverse compounds that possessed drug-like properties and antimicrobial activity against drug-resistant clinical isolates. These data validate ACE as a valuable tool for the fast, efficient detection of specific binding molecules that possess biological activity.

scholarly journals Microfluidic Device for Screening for Target Cell-Specific Binding Molecules by Using Adherent Cells

Micromachines ◽  
2019 ◽  
Vol 10 (1) ◽  
pp. 41 ◽  
Author(s):  
Maho Kaminaga ◽  
Tadashi Ishida ◽  
Tetsuya Kadonosono ◽  
Shinae Kizaka-Kondoh ◽  
Toru Omata
Keyword(s):  
Cancer Cell ◽  
Microfluidic Device ◽  
Target Cell ◽  
Specific Binding ◽  
Growth Factor Receptor ◽  
Target Cells ◽  
Cell Binding ◽  
Pillar Arrays ◽  
Epidermal Growth ◽  
Specific Binding Molecules

This paper proposes a microfluidic device for screening molecules such as aptamers, antibodies, proteins, etc. for target cell-specific binding molecules. The discovery of cancer cell-specific binding molecules was the goal of this study. Its functions include filtering non-target cell-binding molecules, trapping molecules on the surface of target cells, washing away unbound molecules, and collecting target cell-specific binding molecules from target cells. These functions were effectively implemented by using our previously developed micro pillar arrays for cell homogeneous dispersion and pneumatic microvalves for tall microchannels. The device was also equipped with serially connected filter chambers in which non-target cells were cultured to reduce the molecules binding to non-target cells as much as possible. We evaluated the performance of the device using cancer cell lines (N87 cells as target cells and HeLa cells as non-target cells) and two fluorescent dye-labeled antibodies: Anti-human epidermal growth factor receptor 2 (anti-HER2) antibody that binds to target cells and anti-integrin antibody that binds to non-target cells. The results showed that the device could reduce anti-integrin antibodies to the detection limit of fluorescent measurement and collect anti-HER2 antibodies from the target cells.

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