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1دورية أكاديمية
المؤلفون: Cukras, Catherine, Wiley, Henry E, Jeffrey, Brett G, Sen, H Nida, Turriff, Amy, Zeng, Yong, Vijayasarathy, Camasamudram, Marangoni, Dario, Ziccardi, Lucia, Kjellstrom, Sten, Park, Tae Kwon, Hiriyanna, Suja, Wright, J Fraser, Colosi, Peter, Wu, Zhijian, Bush, Ronald A, Wei, Lisa L, Sieving, Paul A
المصدر: Molecular Therapy. 26(9)
مصطلحات موضوعية: Biotechnology, Neurosciences, Clinical Research, Gene Therapy, Clinical Trials and Supportive Activities, Eye Disease and Disorders of Vision, Genetics, Evaluation of treatments and therapeutic interventions, 6.1 Pharmaceuticals, Eye, Adult, Aged, Eye Proteins, Female, Genetic Therapy, Humans, Intravitreal Injections, Male, Middle Aged, Mutation, Retina, Retinoschisis, Young Adult, AAV vector, X-linked retinoschisis, clinical trial, gene therapy, ocular disease, retinal disease, Biological Sciences, Technology, Medical and Health Sciences
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/50r6b7g3Test
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2دورية أكاديمية
المؤلفون: Wright, J. Fraser
المصدر: Molecular Therapy - Methods & Clinical Development ; volume 30, page 332 ; ISSN 2329-0501
مصطلحات موضوعية: Genetics, Molecular Biology, Molecular Medicine
الإتاحة: https://doi.org/10.1016Test/j.omtm.2023.08.001
https://api.elsevier.com/content/article/PII:S2329050123001158?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2329050123001158?httpAccept=text/plainTest -
3دورية أكاديمية
المؤلفون: Wright, J. Fraser
المصدر: Molecular Therapy ; volume 30, issue 6, page 2119-2121 ; ISSN 1525-0016
مصطلحات موضوعية: Drug Discovery, Pharmacology, Genetics, Molecular Biology, Molecular Medicine
الإتاحة: https://doi.org/10.1016Test/j.ymthe.2022.05.004
https://api.elsevier.com/content/article/PII:S1525001622003021?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S1525001622003021?httpAccept=text/plainTest -
4دورية أكاديمية
المؤلفون: San Sebastian, Waldy, Kells, Adrian P, Bringas, John, Samaranch, Lluis, Hadaczek, Piotr, Ciesielska, Agnieszka, Macayan, Michael, Pivirotto, Phillip J, Forsayeth, John, Osborne, Sheryl, Wright, J Fraser, Green, Foad, Heller, Gregory, Bankiewicz, Krystof S
مصطلحات موضوعية: AADC deficiency, AAV2-hAADC, MRI-guided, pressurized infusion
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/6d30b5xgTest
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5دورية أكاديمية
المؤلفون: San Sebastian, Waldy, Kells, Adrian P, Bringas, John, Samaranch, Lluis, Hadaczek, Piotr, Ciesielska, Agnieszka, Macayan, Michael J, Pivirotto, Phillip J, Forsayeth, John, Osborne, Sheryl, Wright, J Fraser, Green, Foad, Heller, Gregory, Bankiewicz, Krystof S, work, two authors contributed equally to this
مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Clinical Research, Biomedical Imaging, Gene Therapy, Genetics, Neurosciences, Brain Disorders, Neurological, AADC deficiency, AAV2-hAADC, MRI-guided, pressurized infusion, Medical biotechnology
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/6d30b5xgTest
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6دورية أكاديمية
المؤلفون: Lattanzi, Annalisa, Camarena, Joab, Lahiri, Premanjali, Segal, Helen, Srifa, Waracharee, Vakulskas, Christopher A., Frock, Richard L., Kenrick, Josefin, Lee, Ciaran, Talbott, Narae, Skowronski, Jason, Cromer, M. Kyle, Charlesworth, Carsten T., Bak, Rasmus O., Mantri, Sruthi, Bao, Gang, DiGiusto, David, Tisdale, John, Wright, J. Fraser, Bhatia, Neehar, Roncarolo, Maria Grazia, Dever, Daniel P., Porteus, Matthew H.
المصدر: Lattanzi , A , Camarena , J , Lahiri , P , Segal , H , Srifa , W , Vakulskas , C A , Frock , R L , Kenrick , J , Lee , C , Talbott , N , Skowronski , J , Cromer , M K , Charlesworth , C T , Bak , R O , Mantri , S , Bao , G , DiGiusto , D , Tisdale , J , Wright , J F , Bhatia , N , Roncarolo , M G , Dever , D P & Porteus , M ....
مصطلحات موضوعية: BONE-MARROW-TRANSPLANTATION, CURE, DOUBLE-STRANDED BREAKS, EFFICIENT, EXPRESSION, HEMOGLOBIN, MIXED CHIMERISM, PROGENITOR CELLS, THERAPY, Reproducibility of Results, Humans, Heterocyclic Compounds, Gene Editing, CRISPR-Cas Systems/genetics, Animals, Anemia, Sickle Cell/genetics, Mice, Hematopoietic Stem Cell Mobilization, Hematopoietic Stem Cells, beta-Globins/genetics
الإتاحة: https://doi.org/10.1126/scitranslmed.abf2444Test
https://pure.au.dk/portal/da/publications/development-of-globin-gene-correction-in-human-hematopoietic-stem-cells-as-a-potential-durable-treatment-for-sickle-cell-diseaseTest(6403e654-be74-4e22-aa96-b83cd5bb4858).html
http://www.scopus.com/inward/record.url?scp=85108359175&partnerID=8YFLogxKTest
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8862191/pdf/nihms-1778457.pdfTest -
7دورية أكاديمية
المؤلفون: Hamilton, Bradley A., Wright, J. Fraser
المصدر: Frontiers in Immunology ; volume 12 ; ISSN 1664-3224
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8دورية أكاديمية
المصدر: Research and Practice in Thrombosis and Haemostasis ; volume 5, issue 6, page e12586 ; ISSN 2475-0379
مصطلحات موضوعية: Hematology
الإتاحة: https://doi.org/10.1002/rth2.12586Test
https://api.elsevier.com/content/article/PII:S2475037922014480?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2475037922014480?httpAccept=text/plainTest -
9دورية أكاديمية
المؤلفون: Ciesielska, Agnieszka, Hadaczek, Piotr, Mittermeyer, Gabriele, Zhou, Shangzhen, Wright, J Fraser, Bankiewicz, Krystof S, Forsayeth, John
المصدر: Molecular Therapy. 21(1)
مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Immunology, Brain Disorders, Genetics, Biotechnology, Gene Therapy, Rare Diseases, Neurosciences, Development of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Inflammatory and immune system, Neurological, Animals, Dependovirus, Genetic Vectors, Immunity, Cellular, Proteins, Rats, Transduction, Genetic, Biological Sciences, Technology, Medical and Health Sciences, Clinical sciences, Medical biotechnology
وصف الملف: application/pdf
الوصول الحر: https://escholarship.org/uc/item/5nv9w2j2Test
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10دورية أكاديمية
المؤلفون: Samelson-Jones, Benjamin J., Finn, Jonathan D., Favaro, Patricia, Wright, J. Fraser, Arruda, Valder R.
المساهمون: NIH/NHLBI
المصدر: Molecular Therapy - Methods & Clinical Development ; volume 17, page 1129-1138 ; ISSN 2329-0501
مصطلحات موضوعية: Genetics, Molecular Biology, Molecular Medicine
الإتاحة: https://doi.org/10.1016Test/j.omtm.2020.05.001
https://api.elsevier.com/content/article/PII:S2329050120300887?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2329050120300887?httpAccept=text/plainTest
