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1دورية أكاديمية
المؤلفون: Ahmed Salman, Ariel Kantor, Michelle E. McClements, Gemma Marfany, Sonia Trigueros, Robert E. MacLaren
المصدر: Pharmaceutics, Vol 14, Iss 9, p 1842 (2022)
مصطلحات موضوعية: CRISPR, gene therapy, retina, non-viral delivery, nanoparticles, AAV, Pharmacy and materia medica, RS1-441
وصف الملف: electronic resource
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2دورية أكاديمية
المؤلفون: Ariel Kantor, Susanne B. Haga
المصدر: Journal of Personalized Medicine, Vol 11, Iss 1, p 45 (2021)
مصطلحات موضوعية: regulation, drugs, precision medicine, expedited review, orphan drugs, Medicine
وصف الملف: electronic resource
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3دورية أكاديمية
المؤلفون: Ariel Kantor, Michelle E. McClements, Robert E. MacLaren
المصدر: International Journal of Molecular Sciences, Vol 21, Iss 17, p 6240 (2020)
مصطلحات موضوعية: CRISPR/Cas9, base-editing, prime-editing, adeno-associated vector, genome engineering, gene therapy, Biology (General), QH301-705.5, Chemistry, QD1-999
وصف الملف: electronic resource
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4
المؤلفون: Alejandro Barrera, Keith Siklenka, Erica Rodriguez, Josephine C. Bodle, Liqing Li, Mariah F. Hazlett, Matthew Gemberling, Anne E. West, Douglas C. Rouse, Isaac B. Hilton, Victoria J. Madigan, Valentina Cigliola, Heather Daniels, Charles A. Gersbach, Luke C. Bartelt, Kenneth D. Poss, Ariel Kantor, Katherine R. Tonn-Eisinger, Fang Liu, Aravind Asokan, Courtney A. Williams, Timothy E. Reddy, Maria Ciofani
المصدر: Nature methods
مصطلحات موضوعية: Male, T-Lymphocytes, Mice, Transgenic, Computational biology, Biology, Biochemistry, Article, Epigenesis, Genetic, Epigenome, Mice, Genetic model, Epigenome editing, CRISPR, Animals, Humans, Guide RNA, Molecular Biology, Epigenomics, Regulation of gene expression, Gene Editing, Cas9, Brain, Cell Biology, Fibroblasts, Gene Expression Regulation, Liver, Female, CRISPR-Cas Systems, Biotechnology
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5
المؤلفون: Ayesha Musa, Robert E MacLaren, Jasmina Cehajic-Kapetanovic, Michelle E. McClements, Ariel Kantor, Kanmin Xue, Joel Quinn
المصدر: Human Gene Therapy
مصطلحات موضوعية: Leber Congenital Amaurosis, Reviews, Cell Cycle Proteins, Computational biology, inherited retinal degenerations, Mice, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Genome editing, Antigens, Neoplasm, Genetics, Animals, Humans, Medicine, CRISPR, Allele, Molecular Biology, Gene, 030304 developmental biology, Gene Editing, 0303 health sciences, Gene knockdown, Cas9, business.industry, Retinal Degeneration, Retinal, Genetic Therapy, Clinical trial, Cytoskeletal Proteins, chemistry, 030220 oncology & carcinogenesis, Molecular Medicine, CRISPR-Cas Systems, business
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d823dc74a5eca261dc228b1ae19a82fe
https://doi.org/10.1089/hum.2020.231 -
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المؤلفون: Robert E MacLaren, Ariel Kantor, Michelle E. McClements, Lewis E Fry, Kanmin Xue, Ruofan Connie Han
المصدر: Expert opinion on orphan drugs
مصطلحات موضوعية: Retinal degeneration, Genetic enhancement, Article, Choroideremia, 03 medical and health sciences, 0302 clinical medicine, Gene replacement, nonsense suppression, medicine, CRISPR, Pharmacology (medical), Pharmacology, Toxicology and Pharmaceutics (miscellaneous), Gene, business.industry, REP1, Health Policy, AAV, medicine.disease, gene therapy, eye diseases, Cell biology, 030220 oncology & carcinogenesis, Rab, antisense oligonucleotides, CHM, business, 030217 neurology & neurosurgery, Intracellular
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7
المؤلفون: Erica Rodriguez, Mariah F. Hazlett, Heather Daniels, Isaac B. Hilton, Anne E. West, Aravind Asokan, Courtney A. Williams, Matthew Gemberling, Charles A. Gersbach, Ariel Kantor, Kenneth D. Poss, Keith Siklenka, Valentina Cigliola, Katherine R. Tonn-Eisinger, Douglas C. Rouse, Fang Liu, Liqing Li, Josephine C. Bodle, Alejandro Barrera, Timothy E. Reddy, Luke C. Bartelt, Victoria J. Madigan, Maria Ciofani
مصطلحات موضوعية: Regulation of gene expression, Cas9, Epigenome editing, CRISPR, Epigenetics, Biology, Fusion protein, Gene, Ex vivo, Cell biology
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::9e49ca7f1b8ea2a06b31e71baa371c63
https://doi.org/10.1101/2021.03.08.434430 -
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المؤلفون: Susanne B. Haga, Ariel Kantor
المصدر: Journal of Personalized Medicine
Volume 11
Issue 1
Journal of Personalized Medicine, Vol 11, Iss 45, p 45 (2021)مصطلحات موضوعية: expedited review, medicine.medical_specialty, Breakthrough therapy, precision medicine, Medicine (miscellaneous), lcsh:Medicine, 030204 cardiovascular system & hematology, Article, drugs, Unmet needs, Orphan drug, 03 medical and health sciences, 0302 clinical medicine, Drug approval, Medicine, 030212 general & internal medicine, Intensive care medicine, business.industry, lcsh:R, regulation, Precision medicine, Priority review, Incentive, orphan drugs, Fast track, business
وصف الملف: application/pdf
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9
المؤلفون: Robert E MacLaren, Ariel Kantor, Lewis E Fry, Kanmin Xue, Ahmed M. Salman, Caroline F Peddle, Jasmina Cehajic-Kapetanovic, Michelle E. McClements
المصدر: Progress in Molecular Biology and Translational Science ISBN: 9780323853019
مصطلحات موضوعية: 0301 basic medicine, Genetic enhancement, Translational medicine, Retinal, Disease, Computational biology, Biology, Genome engineering, Novel gene, 03 medical and health sciences, chemistry.chemical_compound, 030104 developmental biology, 0302 clinical medicine, chemistry, CRISPR, 030217 neurology & neurosurgery
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::5ee8a6ce59a41c330d4e9f44db249dcb
https://doi.org/10.1016/bs.pmbts.2021.01.024 -
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المؤلفون: Michelle E. McClements, Ariel Kantor, Robert E MacLaren
المصدر: International Journal of Molecular Sciences
International Journal of Molecular Sciences, Vol 21, Iss 6240, p 6240 (2020)مصطلحات موضوعية: Genetic enhancement, adeno-associated vector, Computational biology, Review, Biology, Tropism, Catalysis, Genome engineering, lcsh:Chemistry, Inorganic Chemistry, chemistry.chemical_compound, Genome editing, CRISPR, Humans, Point Mutation, Genetic Predisposition to Disease, Vector (molecular biology), Physical and Theoretical Chemistry, Transversion, lcsh:QH301-705.5, Molecular Biology, CRISPR/Cas9, Spectroscopy, Gene Editing, base-editing, Organic Chemistry, General Medicine, Dependovirus, prime-editing, gene therapy, Computer Science Applications, genomic DNA, lcsh:Biology (General), lcsh:QD1-999, chemistry, CRISPR-Cas Systems, genome engineering, DNA, RNA, Guide, Kinetoplastida