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  • 1
    Online Resource
    Online Resource
    Biotechnologies for Gene Therapy : RNA, CRISPR, Nanobots, and Preclinical Applications (2022)
    Cham :Springer International Publishing :
    Details
    Keywords: Medical genetics. ; Biomedical engineering. ; Biotechnology. ; Pharmaceutical chemistry. ; Medical Genetics. ; Biomedical Engineering and Bioengineering. ; Biotechnology. ; Pharmaceutics.
    Description / Table of Contents: 1. Therapeutic potential of microRNAs and their nanoparticle-based delivery in the treatment of liver fibrosis -- 2. MicroRNA as a versatile regulator of Wnt signaling pathway in colorectal cancer -- 3. Small Activating RNA Therapy for Angiogenesis -- 4. Muscular dystrophy therapy using viral vector-based CRISPR/Cas -- 5. CRISPR-Cas based gene therapy to target viral infections -- 6. CRISPR targeting the integrated HTLV-1 virus -- 7. Retroviral vectors for gene therapy of monogenic diseases -- 8. DNA Origami Nanodevices for Therapeutic Delivery Applications.
    Abstract: The purpose of this book is to highlight some of latest developments and applications of CRISPR, RNA, and DNA to treat diseases ranging from cancers to cardiovascular and degenerative disorders. It also features innovations of the delivery methods for nucleic acids ranging from nanodevices made from DNA and pseudo amino acids to viral vectors. This is an ideal book for academics, clinicians, and students interested in gene therapy.
    Type of Medium: Online Resource
    Pages: IX, 203 p. 26 illus., 23 illus. in color. , online resource.
    Edition: 1st ed. 2022.
    ISBN: 9783030933333
    URL: https://doi.org/10.1007/978-3-030-93333-3
    DOI: 10.1007/978-3-030-93333-3
    DDC: 616.042
    Language: English
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  • 2
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    Adaptive evolutionary paths from UV reception to sensing violet light by epistatic interactions (2015)
    American Association for the Advancement of Science (AAAS)
    Details
    Publication Date: 2015-09-20
    Description: Ultraviolet (UV) reception is useful for such basic behaviors as mate choice, foraging, predator avoidance, communication, and navigation, whereas violet reception improves visual resolution and subtle contrast detection. UV and violet reception are mediated by the short wavelength–sensitive (SWS1) pigments that absorb light maximally ( max ) at ~360 nm and ~395 to 440 nm, respectively. Because of strong nonadditive (epistatic) interactions among amino acid changes in the pigments, the adaptive evolutionary mechanisms of these phenotypes are not well understood. Evolution of the violet pigment of the African clawed frog ( Xenopus laevis , max = 423 nm) from the UV pigment in the amphibian ancestor ( max = 359 nm) can be fully explained by eight mutations in transmembrane (TM) I–III segments. We show that epistatic interactions involving the remaining TM IV–VII segments provided evolutionary potential for the frog pigment to gradually achieve its violet-light reception by tuning its color sensitivity in small steps. Mutants in these segments also impair pigments that would cause drastic spectral shifts and thus eliminate them from viable evolutionary pathways. The overall effects of epistatic interactions involving TM IV–VII segments have disappeared at the last evolutionary step and thus are not detectable by studying present-day pigments. Therefore, characterizing the genotype-phenotype relationship during each evolutionary step is the key to uncover the true nature of epistasis.
    Electronic ISSN: 2375-2548
    Topics: Natural Sciences in General
    Published by American Association for the Advancement of Science (AAAS)
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  • 3
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    Amphoteric doping of praseodymium Pr3+ in SrTiO3 grain boundaries (2015)
    American Institute of Physics (AIP)
    Details
    Publication Date: 2015-03-27
    Description: Charge compensation in rare-earth Praseodymium (Pr 3+ ) doped SrTiO 3 plays an important role in determining the overall photoluminescence properties of the system. Here, the Pr 3+ doping behavior in SrTiO 3 grain boundaries (GBs) is analyzed using aberration corrected scanning transmission electron microscopy. The presence of Pr 3+ induces structural variations and changes the statistical prevalence of the GB structures. In contrast to the assumption that Pr 3+ substitutes on the Sr site in the bulk, Pr 3+ is found to substitute on both Sr and Ti sites inside the GBs, with the highest concentration at the Ti sites. This amphoteric doping behavior in the boundary plane is further confirmed by first principles theoretical calculations.
    Print ISSN: 0003-6951
    Electronic ISSN: 1077-3118
    Topics: Physics
    Published by American Institute of Physics (AIP)
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  • 4
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    An improved phase shift reconstruction algorithm of fringe scanning technique for X-ray microscopy (2015)
    American Institute of Physics (AIP)
    Details
    Publication Date: 2015-02-19
    Description: The X-ray phase imaging method has been applied to observe soft biological tissues, and it is possible to image the soft tissues by using the benefit of the so-called “Talbot effect” by an X-ray grating. One type of the X-ray phase imaging method was reported by combining an X-ray imaging microscope equipped by a Fresnel zone plate with a phase grating. Using the fringe scanning technique, a high-precision phase shift image could be obtained by displacing the grating step by step and measuring dozens of sample images. The number of the images was selected to reduce the error caused by the non-sinusoidal component of the Talbot self-image at the imaging plane. A larger number suppressed the error more but increased radiation exposure and required higher mechanical stability of equipment. In this paper, we analyze the approximation error of fringe scanning technique for the X-ray microscopy which uses just one grating and proposes an improved algorithm. We compute the approximation error by iteration and substitute that into the process of reconstruction of phase shift. This procedure will suppress the error even with few sample images. The results of simulation experiments show that the precision of phase shift image reconstructed by the proposed algorithm with 4 sample images is almost the same as that reconstructed by the conventional algorithm with 40 sample images. We also have succeeded in the experiment with real data.
    Print ISSN: 0034-6748
    Electronic ISSN: 1089-7623
    Topics: Electrical Engineering, Measurement and Control Technology , Physics
    Published by American Institute of Physics (AIP)
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  • 5
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    Fast electron flux driven by lower hybrid wave in the scrape-off layer (2015)
    American Institute of Physics (AIP)
    Details
    Publication Date: 2015-02-20
    Description: The fast electron flux driven by Lower Hybrid Wave (LHW) in the scrape-off layer (SOL) in EAST is analyzed both theoretically and experimentally. The five bright belts flowing along the magnetic field lines in the SOL and hot spots at LHW guard limiters observed by charge coupled device and infrared cameras are attributed to the fast electron flux, which is directly measured by retarding field analyzers (RFA). The current carried by the fast electron flux, ranging from 400 to 6000 A/m 2 and in the direction opposite to the plasma current, is scanned along the radial direction from the limiter surface to the position about 25 mm beyond the limiter. The measured fast electron flux is attributed to the high parallel wave refractive index n || components of LHW. According to the antenna structure and the LHW power absorbed by plasma, a broad parallel electric field spectrum of incident wave from the antennas is estimated. The radial distribution of LHW-driven current density is analyzed in SOL based on Landau damping of the LHW. The analytical results support the RFA measurements, showing a certain level of consistency. In addition, the deposition profile of the LHW power density in SOL is also calculated utilizing this simple model. This study provides some fundamental insight into the heating and current drive effects induced by LHW in SOL, and should also help to interpret the observations and related numerical analyses of the behaviors of bright belts and hot spots induced by LHW.
    Print ISSN: 1070-664X
    Electronic ISSN: 1089-7674
    Topics: Physics
    Published by American Institute of Physics (AIP)
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  • 6
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    IBM1, a JmjC domain-containing histone demethylase, is involved in the regulation of RNA-directed DNA methylation through the epigenetic control of RDR2 and DCL3 expression in Arabidopsis (2012)
    Oxford University Press
    Details
    Publication Date: 2012-10-10
    Description: Small RNA-directed DNA methylation (RdDM) is an important epigenetic pathway in Arabidopsis that controls the expression of multiple genes and several developmental processes. RNA-DEPENDENT RNA POLYMERASE 2 (RDR2) and DICER-LIKE 3 (DCL3) are necessary factors in 24-nt small interfering RNA (siRNA) biogenesis, which is part of the RdDM pathway. Here, we found that Increase in BONSAI Methylation 1 (IBM1), a conserved JmjC family histone demethylase, is directly associated with RDR2 and DCL3 chromatin. The mutation of IBM1 induced the hypermethylation of H3K9 and DNA non-CG sites within RDR2 and DCL3 , which repressed their expression. A genome-wide analysis suggested that the reduction in RDR2 and DCL3 expression affected siRNA biogenesis in a locus-specific manner and disrupted RdDM-directed gene repression. Together, our results suggest that IBM1 regulates gene expression through two distinct pathways: direct association to protect genes from silencing by preventing the coupling of histone and DNA methylation, and indirect silencing of gene expression through RdDM-directed repression.
    Print ISSN: 0305-1048
    Electronic ISSN: 1362-4962
    Topics: Biology
    Published by Oxford University Press
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  • 7
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    Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus (2015)
    Oxford University Press
    Details
    Publication Date: 2015-09-25
    Description: The ultimate goal of muscular dystrophy gene therapy is to treat all muscles in the body. Global gene delivery was demonstrated in dystrophic mice more than a decade ago using adeno-associated virus (AAV). However, translation to affected large mammals has been challenging. The only reported attempt was performed in newborn Duchenne muscular dystrophy (DMD) dogs. Unfortunately, AAV injection resulted in growth delay, muscle atrophy and contracture. Here we report safe and bodywide AAV delivery in juvenile DMD dogs. Three ~2-m-old affected dogs received intravenous injection of a tyrosine-engineered AAV-9 reporter or micro-dystrophin (μDys) vector at the doses of 1.92–6.24 x 10 14 viral genome particles/kg under transient or sustained immune suppression. DMD dogs tolerated injection well and their growth was not altered. Hematology and blood biochemistry were unremarkable. No adverse reactions were observed. Widespread muscle transduction was seen in skeletal muscle, the diaphragm and heart for at least 4 months (the end of the study). Nominal expression was detected in internal organs. Improvement in muscle histology was observed in μDys-treated dogs. In summary, systemic AAV gene transfer is safe and efficient in young adult dystrophic large mammals. This may translate to bodywide gene therapy in pediatric patients in the future.
    Print ISSN: 0964-6906
    Electronic ISSN: 1460-2083
    Topics: Biology , Medicine
    Published by Oxford University Press
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  • 8
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    Load and wear experiments on the impact hammer of a vertical shaft impact crusher (2015)
    Institute of Physics (IOP)
    Details
    Publication Date: 2015-12-11
    Description: Impact hammers are important components of impact crushers, and are often shortlived due to the high-impact nature of their use. Wear-resistant alloys are welded to the surface of impact hammers to prolong their service life. In this paper, a simulation model of the rotor and impact hammers in impact crushers was designed to utilize the Discrete Element Method (DEM). The wear-resistant alloy on each impact hammer was divided into twenty-two action regions. The load distribution on each alloy block is affected by the structural and manufacturing parameters of the impact crusher. The wear distribution of the impact hammer was measured by shape morphology according to relative impact crushers. The results demonstrated that the real measurements of wear distribution on the impact hammer were similar to simulated load distribution measurements on the same surface. The study of load distribution of impact hammers by DEM established a theoretical foundation on which to base the optimal ...
    Print ISSN: 1757-8981
    Electronic ISSN: 1757-899X
    Topics: Mechanical Engineering, Materials Science, Production Engineering, Mining and Metallurgy, Traffic Engineering, Precision Mechanics
    Published by Institute of Physics (IOP)
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  • 9
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    The prostate cancer risk variant rs55958994 regulates multiple gene expression through extreme long-range chromatin interaction to control tumor progression (2019)
    American Association for the Advancement of Science (AAAS)
    Details
    Publication Date: 2019
    Description: 〈p〉Genome-wide association studies identified single-nucleotide polymorphism (SNP) rs55958994 as a significant variant associated with increased susceptibility to prostate cancer. However, the mechanisms by which this SNP mediates increased risk to cancer are still unknown. In this study, we show that this variant is located in an enhancer active in prostate cancer cells. Deletion of this enhancer from prostate tumor cells resulted in decreased tumor initiation, tumor growth, and invasive migration, as well as a loss of stem-like cells. Using a combination of capture chromosome conformation capture (Capture-C) and RNA sequencing, we identified genes on the same and different chromosomes as targets regulated by the enhancer. Furthermore, we show that expression of individual candidate target genes in an enhancer-deleted cell line rescued different aspects of tumorigenesis. Our data suggest that the rs55958994-associated enhancer affects prostate cancer progression by influencing expression of multiple genes via long-range chromatin interactions.〈/p〉
    Electronic ISSN: 2375-2548
    Topics: Natural Sciences in General
    Published by American Association for the Advancement of Science (AAAS)
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  • 10
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    Absence of structural transition in M_{0.5}IrTe_{2} (M = Mn, Fe, Co, Ni) (2013)
    American Physical Society (APS)
    Details
    Publication Date: 2013-10-08
    Description: Author(s): J.-Q. Yan, B. Saparov, A. S. Sefat, H. Yang, H. B. Cao, H. D. Zhou, B. C. Sales, and D. G. Mandrus M -doped IrTe 2 ( M  = Mn, Fe, Co, Ni) compounds were synthesized by solid-state reaction. Single crystal x-ray diffraction experiments indicate that part of the doped M ions ( M  = Fe, Co, and Ni) substitute for Ir, and the rest intercalate into the octahedral interstitial sites located in between IrTe 2 ... [Phys. Rev. B 88, 134502] Published Mon Oct 07, 2013
    Keywords: Superfluidity and superconductivity
    Print ISSN: 1098-0121
    Electronic ISSN: 1095-3795
    Topics: Physics
    Published by American Physical Society (APS)
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