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1دورية أكاديمية
المؤلفون: T. V. Egorova, A. A. Piskunov, D. A. Poteryaev
المصدر: Биопрепараты: Профилактика, диагностика, лечение, Vol 24, Iss 2, Pp 123-139 (2024)
مصطلحات موضوعية: gene therapy, gene therapy product, adeno-associated virus, aav, adeno-associated virus vectors, hereditary diseases, haemophilia, duchenne muscular dystrophy, gene therapy efficacy, gene therapy safety, aav production technology, aav modification, triple-plasmid transfection, aav-particle purification, Biotechnology, TP248.13-248.65, Medicine
وصف الملف: electronic resource
العلاقة: https://www.biopreparations.ru/jour/article/view/572Test; https://doaj.org/toc/2221-996XTest; https://doaj.org/toc/2619-1156Test
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2دورية أكاديمية
المؤلفون: Yuji Kashiwakura, Kazuhiro Endo, Atsushi Ugajin, Tomohiro Kikuchi, Shuji Hishikawa, Hitoyasu Nakamura, Yuko Katakai, Nemekhbayar Baatartsogt, Takafumi Hiramoto, Morisada Hayakawa, Nobuhiko Kamoshita, Shoji Yamazaki, Akihiro Kume, Harushi Mori, Naohiro Sata, Yoichi Sakata, Shin-ichi Muramatsu, Tsukasa Ohmori
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 502-514 (2023)
مصطلحات موضوعية: gene therapy, hemophilia B, adeno-associated virus vectors, vector recoverability, intra-hepatic vascular administration, Genetics, QH426-470, Cytology, QH573-671
وصف الملف: electronic resource
العلاقة: http://www.sciencedirect.com/science/article/pii/S2329050123001304Test; https://doaj.org/toc/2329-0501Test
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3دورية أكاديمية
المؤلفون: Ellethy, Abousree T. M., Hassan, Gamal M. A.
المصدر: International Journal of Recent Advances in Multidisciplinary Topics; Vol. 5 No. 4 (2024); 65-72 ; 2582-7839
مصطلحات موضوعية: gene therapy, monogenic and polygenic disorders, CRISPR-Cas9, genome editing delivery system, recombinant adeno-associated virus vectors, exosome, genetic disorders, liver diseases, hemophilia, FDA
وصف الملف: application/pdf
العلاقة: https://journals.ijramt.com/index.php/ijramt/article/view/2889/2905Test; https://journals.ijramt.com/index.php/ijramt/article/view/2889Test
الإتاحة: https://doi.org/10.5281/zenodo.11067723Test
https://journals.ijramt.com/index.php/ijramt/article/view/2889Test -
4دورية أكاديمية
المؤلفون: Christina Wagner, Felix F. Fuchsberger, Bernd Innthaler, Robert Pachlinger, Irene Schrenk, Martin Lemmerer, Ruth Birner-Gruenberger
المصدر: International Journal of Molecular Sciences, Vol 25, Iss 2, p 838 (2024)
مصطلحات موضوعية: adeno-associated virus vectors, nanobodies, magnetic beads, automated mass photometry, analytical ultracentrifugation, Biology (General), QH301-705.5, Chemistry, QD1-999
العلاقة: https://www.mdpi.com/1422-0067/25/2/838Test; https://doaj.org/toc/1661-6596Test; https://doaj.org/toc/1422-0067Test; https://doaj.org/article/e7a74de3ed6943018bb4c9de128e54bbTest
الإتاحة: https://doi.org/10.3390/ijms25020838Test
https://doaj.org/article/e7a74de3ed6943018bb4c9de128e54bbTest -
5دورية أكاديمية
المؤلفون: Benjamin Arnson, Hye Ri Kang, Elizabeth D. Brooks, Dorothy Gheorghiu, Ekaterina Ilich, David Courtney, Jeffrey I. Everitt, Bryan R. Cullen, Dwight D. Koeberl
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 108-119 (2023)
مصطلحات موضوعية: genome editing, CRISPR-Cas9, adeno-associated virus vectors, inherited disorder of metabolism, glycogen storage disease, glucose-6-phosphatase, Genetics, QH426-470, Cytology, QH573-671
وصف الملف: electronic resource
العلاقة: http://www.sciencedirect.com/science/article/pii/S2329050123000372Test; https://doaj.org/toc/2329-0501Test
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6دورية أكاديمية
المؤلفون: Gentaro Tajima, Seoan Huh, Natalie Anne Schmidt, Judith C. Macdonald, Tobias Fleischmann, Keith Merrell Wonnacott
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 74-83 (2022)
مصطلحات موضوعية: genetically modified organisms, environmental risk assessment, virus vectors, adeno-associated virus, viral shedding, multi-regional clinical trials, Genetics, QH426-470, Cytology, QH573-671
وصف الملف: electronic resource
العلاقة: http://www.sciencedirect.com/science/article/pii/S2329050122000778Test; https://doaj.org/toc/2329-0501Test
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7دورية أكاديمية
المؤلفون: Skwiercz Andrzej Tomasz, Zapałowska Anita, Flis Łukasz, Koc-Jurczyk Justyna, Jurczyk Łukasz, Litwińczuk Wojciech, Puchalski Czesław
المصدر: Journal of Horticultural Research, Vol 30, Iss 1, Pp 31-40 (2022)
مصطلحات موضوعية: accessory and occasional nematode species, ecto- and endoparasites, virus vectors, Plant culture, SB1-1110
وصف الملف: electronic resource
العلاقة: https://doaj.org/toc/2353-3978Test
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8دورية أكاديمية
المؤلفون: Papaioannou, Ioannis, Owen, James S, Yáñez-Muñoz, Rafael J
المصدر: International Journal of Experimental Pathology (2023) (In press).
مصطلحات موضوعية: CAR-T cells, adeno-associated virus vectors, antisense oligonucleotides, retrovirus vectors
وصف الملف: text
العلاقة: https://discovery.ucl.ac.uk/id/eprint/10170190/1/Clinical%20applications%20of%20gene%20therapy%20for%20rare%20diseases%20%20A%20review.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10170190Test/
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9دورية أكاديمية
المؤلفون: Arnson, Benjamin, Kang, Hye Ri, Brooks, Elizabeth D., Gheorghiu, Dorothy, Ilich, Ekaterina, Courtney, David, Everitt, Jeffrey I., Cullen, Bryan R., Koeberl, Dwight D.
المصدر: Arnson , B , Kang , H R , Brooks , E D , Gheorghiu , D , Ilich , E , Courtney , D , Everitt , J I , Cullen , B R & Koeberl , D D 2023 , ' Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia ' , Molecular Therapy: Methods and Clinical Development , vol. 29 , pp. 108-119 . https://doi.org/10.1016/j.omtm.2023.03.001Test
مصطلحات موضوعية: von Gierke disease, glucose-6-phosphatase, genome editing, adeno-associated virus vectors, inherited disorder of metabolism, CRISPR-Cas9, glycogen storage disease
وصف الملف: application/pdf
الإتاحة: https://doi.org/10.1016/j.omtm.2023.03.001Test
https://pure.qub.ac.uk/en/publications/8f3a8120-acfc-4d03-8a45-59b2dc280ec1Test
https://pureadmin.qub.ac.uk/ws/files/451480392/Genome_editing_using_Staphylococcus_aureus_Cas9_in_a_canine_model_of_glycogen_storage_disease_Ia.pdfTest -
10دورية أكاديمية
المؤلفون: Christina Wagner, Felix F. Fuchsberger, Bernd Innthaler, Martin Lemmerer, Ruth Birner-Gruenberger
المصدر: International Journal of Molecular Sciences; Volume 24; Issue 13; Pages: 11033
مصطلحات موضوعية: adeno-associated virus vectors, partially filled particles, single-molecule mass photometry, genomic cargo, analytical ultracentrifugation
جغرافية الموضوع: agris
وصف الملف: application/pdf
العلاقة: Molecular Biology; https://dx.doi.org/10.3390/ijms241311033Test