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

العلاقة: https://pure.qub.ac.uk/en/publications/8f3a8120-acfc-4d03-8a45-59b2dc280ec1Test

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

المؤلفون: Koeberl, Dwight D., Koch, Rebecca L., Lim, Jeong‐A., Brooks, Elizabeth D., Arnson, Benjamin D., Sun, Baodong, Kishnani, Priya S.

المصدر: Journal of Inherited Metabolic Disease; Jan2024, Vol. 47 Issue 1, p93-118, 26p

المؤلفون: Han, Sang-oh, Li, Songtao, McCall, Angela, Arnson, Benjamin, Everitt, Jeffrey I., Zhang, Haoyue, Young, Sarah P., ElMallah, Mai K., Koeberl, Dwight D.

المساهمون: National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health

المصدر: Molecular Therapy - Methods & Clinical Development ; volume 17, page 133-142 ; ISSN 2329-0501

مصطلحات موضوعية: Genetics, Molecular Biology, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtm.2019.11.020Test
https://api.elsevier.com/content/article/PII:S2329050119301457?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2329050119301457?httpAccept=text/plainTest

المؤلفون: Han, Sang-Oh, Li, Songtao, McCall, Angela, Arnson, Benjamin, Everitt, Jeffrey I, Zhang, Haoyue, Young, Sarah P, ElMallah, Mai K, Koeberl, Dwight D

مصطلحات موضوعية: Pompe disease, adeno-associated virus vector, glycogen storage disease, infantile

وصف الملف: application/pdf

العلاقة: Molecular therapy. Methods & clinical development; S2329-0501(19)30145-7; https://hdl.handle.net/10161/26519Test

الإتاحة: https://hdl.handle.net/10161/26519Test

المؤلفون: Koeberl, Dwight D., Koch, Rebecca L., Lim, Jeong‐A., Brooks, Elizabeth D., Arnson, Benjamin D., Sun, Baodong, Kishnani, Priya S.

المساهمون: National Institute of Arthritis and Musculoskeletal and Skin Diseases

المصدر: Journal of Inherited Metabolic Disease ; volume 47, issue 1, page 93-118 ; ISSN 0141-8955 1573-2665

الإتاحة: https://doi.org/10.1002/jimd.12654Test

المؤلفون: Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight

المصدر: Molecular Therapy - Methods & Clinical Development ; volume 13, page 431 ; ISSN 2329-0501

مصطلحات موضوعية: Genetics, Molecular Biology, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtm.2019.04.005Test
https://api.elsevier.com/content/article/PII:S2329050119300403?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2329050119300403?httpAccept=text/plainTest

المؤلفون: Han, Sang-Oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight

المساهمون: Duke University School of Medicine Durham, NC, USA, Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), École Pratique des Hautes Études (EPHE), Université Paris Sciences et Lettres (PSL)-Université Paris Sciences et Lettres (PSL)-Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Généthon, Université Pierre et Marie Curie - Paris 6 (UPMC), Department of Molecular Genetics and Microbiology Durham, Duke University Durham

المصدر: EISSN: 2329-0501 ; Molecular Therapy - Methods and Clinical Development ; https://hal.sorbonne-universite.fr/hal-01432085Test ; Molecular Therapy - Methods and Clinical Development, 2017, ⟨10.1016/j.omtm.2016.12.010⟩

مصطلحات موضوعية: [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology

العلاقة: hal-01432085; https://hal.sorbonne-universite.fr/hal-01432085Test; https://hal.sorbonne-universite.fr/hal-01432085/documentTest; https://hal.sorbonne-universite.fr/hal-01432085/file/1-s2.0-S2329050117300049-main.pdfTest

الإتاحة: https://doi.org/10.1016/j.omtm.2016.12.010Test
https://hal.sorbonne-universite.fr/hal-01432085Test
https://hal.sorbonne-universite.fr/hal-01432085/documentTest
https://hal.sorbonne-universite.fr/hal-01432085/file/1-s2.0-S2329050117300049-main.pdfTest

المؤلفون: Arnson, Benjamin Donald

المساهمون: Koeberl, Dwight D, Cullen, Bryan R

مصطلحات موضوعية: Genetics, Molecular biology, Microbiology, Adeno-Associated Virus, CRISPR/Cas9, Gene Therapy, Genome Editing, Glucose-6-Phosphatase, Glycogen Storage Disease

وصف الملف: application/pdf

العلاقة: https://hdl.handle.net/10161/27759Test

الإتاحة: https://hdl.handle.net/10161/27759Test