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1

Nuno-Gonzalez, Patricia, Hsu Chao, and Kazuhiro Oka. "Targeting site-specific chromosome integration." Acta Biochimica Polonica 52, no. 2 (2005): 285–91. http://dx.doi.org/10.18388/abp.2005_3441.

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The concept of gene therapy was introduced with great promise and high expectations. However, what appeared simple in theory has not translated into practice. Despite some success in clinical trials, the research community is still facing an old problem: namely, the need for a vector that can deliver a gene to target cells without adverse events while maintaining a long-term therapeutic effect. Some of these challenges are being addressed by the development of hybrid vectors which meld two different viral systems to incorporate efficient gene delivery and large cloning capacity with site-speci
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Horn, C., and A. M. Handler. "Site-specific genomic targeting in Drosophila." Proceedings of the National Academy of Sciences 102, no. 35 (2005): 12483–88. http://dx.doi.org/10.1073/pnas.0504305102.

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Fruitwala, Mushtaq A., and N. M. Sanghavi. "Site-Specific Drug Targeting with Fluorouracil Microspheres." Drug Delivery 3, no. 1 (1996): 5–8. http://dx.doi.org/10.3109/10717549609031375.

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García-Otín, Angel-Luis. "Mammalian genome targeting using site-specific recombinases." Frontiers in Bioscience 11, no. 1 (2006): 1108. http://dx.doi.org/10.2741/1867.

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5

Tomlinson, E. "Site-Specific Drug Carriers." Engineering in Medicine 15, no. 4 (1986): 197–202. http://dx.doi.org/10.1243/emed_jour_1986_015_053_02.

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Site-specific drug carriers are required to exclusively deliver drug molecules to difficult targets within the body. They should do so in a form which protects the drug and host from one another. This contribution reviews the reasons for drug targeting, and describes some of the features required of two types of carrier system, i.e., particulates and soluble (bio)conjugates.
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D'Souza, Martin J., and Patrick DeSouza. "Site specific microencapsulated drug targeting strategies- liver and gastro-intestinal tract targeting." Advanced Drug Delivery Reviews 17, no. 3 (1995): 247–54. http://dx.doi.org/10.1016/0169-409x(95)00058-f.

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Mason, Rosemarie, Cameron Adams, Carole Bewley, John Mascola, and Mario Roederer. "Potent SIV-specific Antibodies Targeting the Cyanovirin Binding Site." AIDS Research and Human Retroviruses 30, S1 (2014): A211—A212. http://dx.doi.org/10.1089/aid.2014.5457c.abstract.

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Gorman, Cori, and Clayton Bullock. "Site-specific gene targeting for gene expression in eukaryotes." Current Opinion in Biotechnology 11, no. 5 (2000): 455–60. http://dx.doi.org/10.1016/s0958-1669(00)00127-0.

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Lovering, Richard M., Camilo Vanegas, Stephen J. P. Pratt, Su Xu, and Jason Hammond. "Site-specific Targeting Platelet-rich Plasma Via Superparamagnetic Nanoparticles." Medicine & Science in Sports & Exercise 46 (May 2014): 357. http://dx.doi.org/10.1249/01.mss.0000494250.63191.70.

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10

Bodor, N. "Drag targeting by site-specific chemical drug delivery systems." European Journal of Pharmacology 183, no. 1 (1990): 119–20. http://dx.doi.org/10.1016/0014-2999(90)91385-o.

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Metzger, Daniel, and Pierre Chambon. "Site- and Time-Specific Gene Targeting in the Mouse." Methods 24, no. 1 (2001): 71–80. http://dx.doi.org/10.1006/meth.2001.1159.

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12

Wigley, P., C. Becker, J. Beltrame, et al. "Site-specific transgene insertion: an approach." Reproduction, Fertility and Development 6, no. 5 (1994): 585. http://dx.doi.org/10.1071/rd9940585.

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Methods to improve the production of transgenic animals are being developed. Conventional transgenesis, involving microinjection of DNA into fertilized eggs, has a number of limitations. These result from the inability to control both the site of transgene insertion and the number of gene copies inserted. The approach described seeks to overcome these problems and to allow single copy insertion of transgenes into a defined site in animal genomes. The method involves the use of embryonic stem cells, gene targeting and the FLP recombinase system.
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13

De Rosa, Lucia, Rossella Di Stasi, Alessandra Romanelli, and Luca Domenico D’Andrea. "Exploiting Protein N-Terminus for Site-Specific Bioconjugation." Molecules 26, no. 12 (2021): 3521. http://dx.doi.org/10.3390/molecules26123521.

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Although a plethora of chemistries have been developed to selectively decorate protein molecules, novel strategies continue to be reported with the final aim of improving selectivity and mildness of the reaction conditions, preserve protein integrity, and fulfill all the increasing requirements of the modern applications of protein conjugates. The targeting of the protein N-terminal alpha-amine group appears a convenient solution to the issue, emerging as a useful and unique reactive site universally present in each protein molecule. Herein, we provide an updated overview of the methodologies
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14

Bolzati, Cristina, and Barbara Spolaore. "Enzymatic Methods for the Site-Specific Radiolabeling of Targeting Proteins." Molecules 26, no. 12 (2021): 3492. http://dx.doi.org/10.3390/molecules26123492.

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Site-specific conjugation of proteins is currently required to produce homogenous derivatives for medicine applications. Proteins derivatized at specific positions of the polypeptide chain can actually show higher stability, superior pharmacokinetics, and activity in vivo, as compared with conjugates modified at heterogeneous sites. Moreover, they can be better characterized regarding the composition of the derivatization sites as well as the conformational and activity properties. To this aim, several site-specific derivatization approaches have been developed. Among these, enzymes are powerf
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15

Talaie, Tara, Stephen J. P. Pratt, Camilo Vanegas, et al. "Site-Specific Targeting of Platelet-Rich Plasma via Superparamagnetic Nanoparticles." Orthopaedic Journal of Sports Medicine 3, no. 1 (2015): 232596711456618. http://dx.doi.org/10.1177/2325967114566185.

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Natarajan, Arutselvan, Cheng-Yi Xiong, Huguette Albrecht, Gerald L. DeNardo, and Sally J. DeNardo. "Characterization of Site-Specific ScFv PEGylation for Tumor-Targeting Pharmaceuticals." Bioconjugate Chemistry 16, no. 1 (2005): 113–21. http://dx.doi.org/10.1021/bc0498121.

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17

Yun, Yang H., Douglas J. Goetz, Paige Yellen, and Weiliam Chen. "Hyaluronan microspheres for sustained gene delivery and site-specific targeting." Biomaterials 25, no. 1 (2004): 147–57. http://dx.doi.org/10.1016/s0142-9612(03)00467-8.

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Varbanov, Petar Sabev, Zsófia Fodor та Jiří Jaromír Klemeš. "Total Site targeting with process specific minimum temperature difference (ΔTmin)". Energy 44, № 1 (2012): 20–28. http://dx.doi.org/10.1016/j.energy.2011.12.025.

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Alawieh, Ali, and Stephen Tomlinson. "Injury site-specific targeting of complement inhibitors for treating stroke." Immunological Reviews 274, no. 1 (2016): 270–80. http://dx.doi.org/10.1111/imr.12470.

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Mishra, Abhinav P., Suresh Chandra, Ruchi Tiwari, Ashish Srivastava, and Gaurav Tiwari. "Therapeutic Potential of Prodrugs Towards Targeted Drug Delivery." Open Medicinal Chemistry Journal 12, no. 1 (2018): 111–23. http://dx.doi.org/10.2174/1874104501812010111.

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In designing of Prodrugs, targeting can be achieved in two ways: site-specified drug delivery and site-specific drug bioactivation. Prodrugs can be designed to target specific enzymes or carriers by considering enzyme-substrate specificity or carrier-substrate specificity in order to overcome various undesirable drug properties. There are certain techniques which are used for tumor targeting such as Antibody Directed Enzyme Prodrug Therapy [ADEPT] Gene-Directed Enzyme Prodrug Therapy [GDEPT], Virus Directed Enzyme Prodrug Therapy [VDEPT] and Gene Prodrug Activation Therapy [GPAT]. Our review f
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21

Chen, Lanxi, Jianhua Zhou, Qiurong Deng, et al. "Ultrasound-visualized, site-specific vascular embolization using magnetic protein microcapsules." Journal of Materials Chemistry B 9, no. 10 (2021): 2407–16. http://dx.doi.org/10.1039/d0tb02715d.

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A novel embolization strategy combining ultrasound visualization and magnetic targeting functions was developed using the fabricated magnetic protein microcapsules (MPMs) and holds great potential in the treatment of hepatocellular carcinoma (HCC).
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22

Ito, Masanori, Keitaro Yamanouchi, Kunihiko Naito, Michele P. Calos, and Hideaki Tojo. "Site-specific integration of transgene targeting an endogenous lox-like site in early mouse embryos." Journal of Applied Genetics 52, no. 1 (2010): 89–94. http://dx.doi.org/10.1007/s13353-010-0011-3.

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23

Neerman, M. "Enhancing the Site-Specific Targeting of Macromolecular Anticancer Drug Delivery Systems." Current Drug Targets 7, no. 2 (2006): 229–35. http://dx.doi.org/10.2174/138945006775515473.

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24

Marjomaki, V., T. Lahtinen, M. Martikainen, et al. "Site-specific targeting of enterovirus capsid by functionalized monodisperse gold nanoclusters." Proceedings of the National Academy of Sciences 111, no. 4 (2014): 1277–81. http://dx.doi.org/10.1073/pnas.1310973111.

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25

Sesardic, D., V. Khan, and M. J. Corbel. "Targeting of specific domains of diphtheria toxin by site-directed antibodies." Journal of General Microbiology 138, no. 10 (1992): 2197–203. http://dx.doi.org/10.1099/00221287-138-10-2197.

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26

Palchaudhuri, Rahul, Kwan-Keat Ang, Borja Saez, David B. Sykes, Gregory L. Verdine, and David T. Scadden. "Differentiation Induction In Acute Myeloid Leukemia Using Site-Specific DNA-Targeting." Blood 122, no. 21 (2013): 3940. http://dx.doi.org/10.1182/blood.v122.21.3940.3940.

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Abstract Hoxa9 and Meis1 are overexpressed in >70% of acute myeloid leukemia (AML) and associated with poor prognosis and survival. Hoxa9 and Meis1 interact with DNA and PBX to achieve transcription of differentiation-blocking genes. We tested transcriptional repression at Hoxa9-PBX-Meis1 genomic binding sites to induce differentiation in a model of human AML We designed a DNA-recognition strategy based on the known structure of the Hoxa9-PBX-DNA complex by fusing the DNA binding helices of Hoxa9 and PBX to create concise homeodomain fusion proteins that target the Hoxa9-PBX DNA recognition
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27

Polyak, Boris, and Gary Friedman. "Magnetic targeting for site-specific drug delivery: applications and clinical potential." Expert Opinion on Drug Delivery 6, no. 1 (2009): 53–70. http://dx.doi.org/10.1517/17425240802662795.

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28

Babincová, Melánia, Veronika Altanerová, Miloš Lampert, et al. "Site-Specific in vivo Targeting of Magnetoliposomes Using Externally Applied Magnetic Field." Zeitschrift für Naturforschung C 55, no. 3-4 (2000): 278–81. http://dx.doi.org/10.1515/znc-2000-3-422.

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Abstract Human serum albumin labeled with technetium-99m was encapsulated together with magnetite particles into phosphatidylcholine/cholesterol liposomes. In order to investigate the stability of this complex and its ability to be used for magnetic drug targeting, the in-vivo distribution after intravenous administration in rats was estimated. For in-vivo targeting an SmCo permanent magnet with intensity ~ 0.3 5 T was attached near the right kidney. Difference between the relative radioactivity in the magnetically targeted right kidney (25.92±5.84%) and non-targeted left kidney (0.93±0.05%) i
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29

Hwang, Dobeen, and Christoph Rader. "Site-Specific Antibody–Drug Conjugates in Triple Variable Domain Fab Format." Biomolecules 10, no. 5 (2020): 764. http://dx.doi.org/10.3390/biom10050764.

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The interest in replacing the conventional immunoglobulin G (IgG) format of monoclonal antibodies (mAbs) and antibody–drug conjugates (ADCs) with alternative antibody and antibody-like scaffolds reflects a need to expand their therapeutic utility and potency while retaining their exquisite specificity, affinity, and low intrinsic toxicity. For example, in the therapy of solid malignancies, the limited tumor tissue penetration and distribution of ADCs in IgG format mitigates a uniform distribution of the cytotoxic payload. Here, we report triple variable domain Fab (TVD–Fab) as a new format tha
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30

Rizzuto, Gabriella, Barbara Gorgoni, Manuela Cappelletti, et al. "Development of Animal Models for Adeno-Associated Virus Site-Specific Integration." Journal of Virology 73, no. 3 (1999): 2517–26. http://dx.doi.org/10.1128/jvi.73.3.2517-2526.1999.

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ABSTRACT The adeno-associated virus (AAV) is unique in its ability to target viral DNA integration to a defined region of human chromosome 19 (AAVS1). Since AAVS1 sequences are not conserved in a rodent’s genome, no animal model is currently available to study AAV-mediated site-specific integration. We describe here the generation of transgenic rats and mice that carry the AAVS1 3.5-kb DNA fragment. To test the response of the transgenic animals to Rep-mediated targeting, primary cultures of mouse fibroblasts, rat hepatocytes, and fibroblasts were infected with wild-type wt AAV. PCR amplificat
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31

Zhen-Dan, Shi, Li Wan-Li, Zhang Yong-Liang, and Chen Xue-Jin. "Advances in the development of animal gene transfer." Chinese Journal of Agricultural Biotechnology 5, no. 2 (2008): 101–6. http://dx.doi.org/10.1017/s1479236208002179.

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AbstractEfficiency and specificity are key limiting factors for the production of transgenic animals. This review describes the recently developed animal gene transfer techniques, including non-site-specific methods of gene transfer into the testis and ovary for easy production of transgenic animals; gene targeting in embryonic stem cells, somatic cells and primordial germ cells for site-specific methods; methods to improve cloning efficiency in gene targeting; and site- and timing-specific gene targeting and controlled expression of transferred genes. In addition, methods of utilizing newly d
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32

Lowder, Leah L., Matthew Powell, Sean E. Miller, et al. "Mechanistic Investigation of Site-specific DNA Methylating Agents Targeting Breast Cancer Cells." Journal of Medicinal Chemistry 64, no. 17 (2021): 12651–69. http://dx.doi.org/10.1021/acs.jmedchem.1c00615.

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Enz, Doris. "GABA(A) receptor subtype-selective loreclezole analogues targeting an α6-specific site". Intrinsic Activity 7, Suppl. 1 (2019): A3.12. http://dx.doi.org/10.25006/ia.7.s1-a3.12.

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Möhlmann, Sina, Peter Bringmann, Simone Greven, and Axel Harrenga. "Site-specific modification of ED-B-targeting antibody using intein-fusion technology." BMC Biotechnology 11, no. 1 (2011): 76. http://dx.doi.org/10.1186/1472-6750-11-76.

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Deng, R., X. F. Zhu, Y. Huang, and W. Q. Zhong. "293 (PB281): Targeting site-specific N-glycosylated B7H3 induces potent antitumor immunity." European Journal of Cancer 211 (October 2024): 114807. http://dx.doi.org/10.1016/j.ejca.2024.114807.

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36

Merrihew, Raymond V., R. Geoffrey Sargent, and John H. Wilson. "Efficient modification of the APRT gene by FLP/FRT site-specific targeting." Somatic Cell and Molecular Genetics 21, no. 5 (1995): 299–307. http://dx.doi.org/10.1007/bf02257465.

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van Berkel, Th J. C. "Drug targeting: application of endogenous carriers for site-specific delivery of drugs." Journal of Controlled Release 24, no. 1-3 (1993): 145–55. http://dx.doi.org/10.1016/0168-3659(93)90174-4.

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Szulc, Zdzislaw M., Jacek Bielawski, Hanna Gracz, et al. "Tailoring structure–function and targeting properties of ceramides by site-specific cationization." Bioorganic & Medicinal Chemistry 14, no. 21 (2006): 7083–104. http://dx.doi.org/10.1016/j.bmc.2006.07.016.

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39

Albone, E., X. Cheng, A. Verdi, et al. "579P MORAb-109: A site-specific eribulin-conjugated ADC targeting human mesothelin." Annals of Oncology 31 (September 2020): S491—S492. http://dx.doi.org/10.1016/j.annonc.2020.08.693.

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40

Simon, Johanna, Michael Fichter, Gabor Kuhn, et al. "Achieving dendritic cell subset-specific targeting in vivo by site-directed conjugation of targeting antibodies to nanocarriers." Nano Today 43 (April 2022): 101375. http://dx.doi.org/10.1016/j.nantod.2022.101375.

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Agrawal, Shivanshu, Anuj Garg, and Vikas Varshney. "Recent Updates On Applications of Lipid-Based Nanoparticles For Site- Specific Drug Delivery." Pharmaceutical Nanotechnology 10, no. 1 (2022): 24–41. http://dx.doi.org/10.2174/2211738510666220304111848.

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Background: Site-specific drug delivery is a widespread and demanding area nowadays. Lipid-based nanoparticulate drug delivery systems have shown promising effects for targeting drugs among lymphatic systems, brain tissues, lungs, and skin. Recently, lipid nanoparticles are used for targeting the brain via the mucosal route for local therapeutic effects. Lipid nanoparticles (LNPs) can help in enhancing the efficacy and lowering the toxicities of anticancer drugs to treat the tumors, particularly in lymph after metastases of tumors. LNPs contain a non-polar core that can improve the absorption
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Italia, James Sebastian, Nikos Biris, Zhi Li, Myer Hussain, John Boyce, and Audrey Warner. "Abstract 1767: A next generation site-specific ADC targeting breast and gastric cancer." Cancer Research 82, no. 12_Supplement (2022): 1767. http://dx.doi.org/10.1158/1538-7445.am2022-1767.

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Abstract Antibody drug conjugates are entering a renaissance period as a promising treatment option for a variety of cancers. Despite some success in the past few decades, developing effective ADCs remains a challenge due to extensive inefficiencies of industry standard conjugation technologies. Industry available methods can be limited by lack of site-specificity, inflexibility on the site of conjugation, and poor overall biophysical characteristics which can alter the efficacy, safety, and bioavailability of these therapeutics. BrickBio’s unique bioconjugation methodology enables precise (si
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43

McGeary, Sean E., Kathy S. Lin, Charlie Y. Shi, et al. "The biochemical basis of microRNA targeting efficacy." Science 366, no. 6472 (2019): eaav1741. http://dx.doi.org/10.1126/science.aav1741.

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MicroRNAs (miRNAs) act within Argonaute proteins to guide repression of messenger RNA targets. Although various approaches have provided insight into target recognition, the sparsity of miRNA-target affinity measurements has limited understanding and prediction of targeting efficacy. Here, we adapted RNA bind-n-seq to enable measurement of relative binding affinities between Argonaute-miRNA complexes and all sequences ≤12 nucleotides in length. This approach revealed noncanonical target sites specific to each miRNA, miRNA-specific differences in canonical target-site affinities, and a 100-fold
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44

Chaves, Lorena C. S., Nichole Orr-Burks, Daryll Vanover, et al. "mRNA-encoded Cas13 treatment of Influenza via site-specific degradation of genomic RNA." PLOS Pathogens 20, no. 7 (2024): e1012345. http://dx.doi.org/10.1371/journal.ppat.1012345.

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The CRISPR-Cas13 system has been proposed as an alternative treatment of viral infections. However, for this approach to be adopted as an antiviral, it must be optimized until levels of efficacy rival or exceed the performance of conventional approaches. To take steps toward this goal, we evaluated the influenza viral RNA degradation patterns resulting from the binding and enzymatic activity of mRNA-encoded LbuCas13a and two crRNAs from a prior study, targeting PB2 genomic and messenger RNA. We found that the genome targeting guide has the potential for significantly higher potency than origin
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Hristova-Panusheva, Kamelia, Charilaos Xenodochidis, Milena Georgieva, and Natalia Krasteva. "Nanoparticle-Mediated Drug Delivery Systems for Precision Targeting in Oncology." Pharmaceuticals 17, no. 6 (2024): 677. http://dx.doi.org/10.3390/ph17060677.

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Nanotechnology has emerged as a transformative force in oncology, facilitating advancements in site-specific cancer therapy and personalized oncomedicine. The development of nanomedicines explicitly targeted to cancer cells represents a pivotal breakthrough, allowing the development of precise interventions. These cancer-cell-targeted nanomedicines operate within the intricate milieu of the tumour microenvironment, further enhancing their therapeutic efficacy. This comprehensive review provides a contemporary perspective on precision cancer medicine and underscores the critical role of nanotec
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46

Krafčík, A., P. Babinec, and M. Babincová. "Feasibility of subcutaneously implanted magnetic microarrays for site specific drug and gene targeting." Journal of Engineering Science and Technology Review 3, no. 1 (2010): 53–57. http://dx.doi.org/10.25103/jestr.031.10.

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Liang, Xin, Susan R. Russell, Sandra Estelle, et al. "Specific Inhibition Of Ectodomain Shedding Of GPIba By Targeting Its Shedding Cleavage Site." Blood 122, no. 21 (2013): 21. http://dx.doi.org/10.1182/blood.v122.21.21.21.

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Abstract Background Ectodomain shedding of GPIbα, a proteolytic event in which metalloprotease ADAM17 cleaves the Gly464-Val465 bond and releases glycocalicin to the plasma, is considered a critical step in mediating clearance of stored platelets. Supporting evidence has been obtained from animal studies using ADAM17 inhibitors. However, the definitive proof is lacking due to the broad substrate specificity of ADAM17. We report herein novel monoclonal antibodies (MAbs) that specifically inhibit shedding of human GPIbα in platelets and may be potentially developed into an additive to improve pl
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48

Yoda, Hiroyuki, Takahiro Inoue, Yoshinao Shinozaki, et al. "Direct Targeting of MYCN Gene Amplification by Site-Specific DNA Alkylation in Neuroblastoma." Cancer Research 79, no. 4 (2018): 830–40. http://dx.doi.org/10.1158/0008-5472.can-18-1198.

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Ng, Philip, and Mark D. Baker. "High efficiency site-specific modification of the chromosomal immunoglobulin locus by gene targeting." Journal of Immunological Methods 214, no. 1-2 (1998): 81–96. http://dx.doi.org/10.1016/s0022-1759(98)00033-7.

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F. Kolb, Andreas, and Stuart G. Siddell. "Genomic targeting of a bicistronic DNA fragment by Cre-mediated site-specific recombination." Gene 203, no. 2 (1997): 209–16. http://dx.doi.org/10.1016/s0378-1119(97)00515-5.

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