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

الوصول الحر: https://doaj.org/article/8282e120c0924cec897cf976a2665255Test

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

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 133-142 (2020)

مصطلحات موضوعية: Pompe disease, glycogen storage disease, adeno-associated virus vector, infantile, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

العلاقة: http://www.sciencedirect.com/science/article/pii/S2329050119301457Test; https://doaj.org/toc/2329-0501Test

الوصول الحر: https://doaj.org/article/c420a65b4c434febb94fb8a86044fc55Test

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

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 4, Iss C, Pp 126-136 (2017)

مصطلحات موضوعية: gene therapy, adeno-associated virus, acid α-glucosidase, acid maltase, glycogen storage disease type II, immune tolerance induction, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

العلاقة: http://www.sciencedirect.com/science/article/pii/S2329050117300049Test; https://doaj.org/toc/2329-0501Test

الوصول الحر: https://doaj.org/article/9139c3ce594942769f879f7b29b34fedTest

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

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 133-142 (2020)
Molecular Therapy. Methods & Clinical Development

مصطلحات موضوعية: 0301 basic medicine, congenital, hereditary, and neonatal diseases and abnormalities, lcsh:QH426-470, viruses, Genetic enhancement, Disease, Biology, Genome, Article, Virus, adeno-associated virus vector, 03 medical and health sciences, Transduction (genetics), 0302 clinical medicine, glycogen storage disease, Glycogen storage disease type II, Genetics, medicine, Glycogen storage disease, lcsh:QH573-671, Molecular Biology, infantile, lcsh:Cytology, Pompe disease, nutritional and metabolic diseases, medicine.disease, Virology, 3. Good health, lcsh:Genetics, 030104 developmental biology, 030220 oncology & carcinogenesis, Knockout mouse, Molecular Medicine

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f2e9e473719d511180f28d63324ee43cTest
https://doi.org/10.1016/j.omtm.2019.11.020Test

المؤلفون: Songtao Li, Sang-oh Han, Jeffrey I. Everitt, Angela L McCall, Dwight D. Koeberl, Benjamin Arnson, Mai K. ElMallah

المصدر: Molecular Genetics and Metabolism. 129:S69-S70

مصطلحات موضوعية: Endocrinology, Depot, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Genetics, Physiology, Medicine, Disease, business, Molecular Biology, Biochemistry

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::1773ba2e8bacd641022ab6cf5ba120c3Test
https://doi.org/10.1016/j.ymgme.2019.11.165Test

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

المساهمون: HAL-UPMC, Gestionnaire, Duke University School of Medicine, 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], École Pratique des Hautes Études (EPHE)

المصدر: Molecular Therapy-Methods and Clinical Development
Molecular Therapy-Methods and Clinical Development, 2017, ⟨10.1016/j.omtm.2016.12.010⟩
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 4, Iss C, Pp 126-136 (2017)
Molecular Therapy-Methods and Clinical Development, Nature Publishing Group, 2017, ⟨10.1016/j.omtm.2016.12.010⟩

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, lcsh:QH426-470, Dose, Genetic enhancement, adeno-associated virus, Biology, medicine.disease_cause, Article, Immune tolerance, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Internal medicine, Glycogen storage disease type II, Genetics, medicine, lcsh:QH573-671, Molecular Biology, Adeno-associated virus, 030304 developmental biology, immune tolerance induction, 0303 health sciences, [SDV.MHEP] Life Sciences [q-bio]/Human health and pathology, Glycogen, lcsh:Cytology, nutritional and metabolic diseases, Enzyme replacement therapy, medicine.disease, gene therapy, 3. Good health, lcsh:Genetics, 030104 developmental biology, Endocrinology, chemistry, acid maltase, glycogen storage disease type II, 030220 oncology & carcinogenesis, acid α-glucosidase, biology.protein, Molecular Medicine, Original Article, Antibody, [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::49e24a33b98717a9fbfbfe3572487475Test
https://hal.sorbonne-universite.fr/hal-01432085/documentTest