المؤلفون: Anthony A. Stephenson, Stefan Nicolau, Tatyana A. Vetter, Gabrielle P. Dufresne, Emma C. Frair, Jessica E. Sarff, Gregory L. Wheeler, Benjamin J. Kelly, Peter White, Kevin M. Flanigan

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 486-499 (2023)

مصطلحات موضوعية: Duchenne muscular dystrophy, dystrophin, exon 2 duplication, gene editing, CRISPR-Cas9, homology-independent targeted integration, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050123001237; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/3a64dafda5294680b6d821cf373f9511

المؤلفون: Liubov V. Gushchina, Adrienne J. Bradley, Tatyana A. Vetter, Jacob W. Lay, Natalie L. Rohan, Emma C. Frair, Nicolas Wein, Kevin M. Flanigan

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 31, Iss , Pp 101144- (2023)

مصطلحات موضوعية: Duchenne muscular dystrophy, Becker muscular dystrophy, dystrophin, exon skipping, U7snRNA, scAAV9.U7.ACCA, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050123001833; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/1dee77955bc4458f84bf563a278aee74

المؤلفون: Kevin M. Flanigan, Tatyana A. Vetter, Tabatha R. Simmons, Megan Iammarino, Emma C. Frair, Federica Rinaldi, Louis G. Chicoine, Johan Harris, John P. Cheatham, Sharon L. Cheatham, Brian Boe, Megan A. Waldrop, Deborah A. Zygmunt, Davin Packer, Paul T. Martin

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 47-60 (2022)

مصطلحات موضوعية: Duchenne muscular dystrophy, AAV, gene therapy, GALGT2, B4GALNT, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S232905012200119X; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/5d4cb4d3fc0f4849bd3ccf973f1e61e1

المؤلفون: Liubov V. Gushchina, Tatyana A. Vetter, Emma C. Frair, Adrienne J. Bradley, Kelly M. Grounds, Jacob W. Lay, Nianyuan Huang, Aisha Suhaiba, Frederick J. Schnell, Gunnar Hanson, Tabatha R. Simmons, Nicolas Wein, Kevin M. Flanigan

المصدر: Molecular Therapy: Nucleic Acids, Vol 30, Iss , Pp 479-492 (2022)

مصطلحات موضوعية: MT: oligonucleotides: therapies and applications, Duchenne muscular dystrophy, DMD, dystrophin, phosphorodiamidate morpholino oligomers, PMO, cell-penetrating peptide-conjugated PMO, PPMO, antisense oligonucleotide, AO, Therapeutics. Pharmacology, RM1-950

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253122002918; https://doaj.org/toc/2162-2531

URL الوصول: https://doaj.org/article/c58f1dc6613c474f8ea734b55a626d47

المؤلفون: Nicolas Wein, Tatyana A. Vetter, Adeline Vulin, Tabatha R. Simmons, Emma C. Frair, Adrienne J. Bradley, Liubov V. Gushchina, Camila F. Almeida, Nianyuan Huang, Daniel Lesman, Dhanarajan Rajakumar, Robert B. Weiss, Kevin M. Flanigan

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 279-293 (2022)

مصطلحات موضوعية: Duchenne muscular dystrophy, Becker muscular dystrophy, dystrophin, exon skipping, U7snRNA, gene therapy, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050122000973; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/937ffb28cf15459caaa9793b8cebec7b

المؤلفون: Michelle Eggers, Nicolas Wein, Hemantkumar D Chavan, Kevin M. Flanigan, Adrienne J Bradley, Liubov V Gushchina, Emma C Frair, Hsin-Jung Chou, Megan A. Waldrop, Tabatha R. Simmons, Natalie Rohan

المصدر: Human Gene Therapy. 32:882-894

مصطلحات موضوعية: musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities, 0303 health sciences, business.industry, Duchenne muscular dystrophy, medicine.disease, medicine.disease_cause, Exon skipping, 03 medical and health sciences, Exon, 0302 clinical medicine, 030220 oncology & carcinogenesis, Toxicity, Genetics, medicine, Cancer research, Molecular Medicine, Vector (molecular biology), business, Molecular Biology, Adeno-associated virus, 030304 developmental biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::5e6a19be41010c71837d9b049f88e3ad
https://doi.org/10.1089/hum.2020.286

المؤلفون: Kevin M, Flanigan, Tatyana A, Vetter, Tabatha R, Simmons, Megan, Iammarino, Emma C, Frair, Federica, Rinaldi, Louis G, Chicoine, Johan, Harris, John P, Cheatham, Sharon L, Cheatham, Brian, Boe, Megan A, Waldrop, Deborah A, Zygmunt, Davin, Packer, Paul T, Martin

المصدر: Molecular therapy. Methodsclinical development. 27

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid________::df1ab05d52fafd3eb7edbd550bd3b3be
https://pubmed.ncbi.nlm.nih.gov/36186954

المؤلفون: Adrienne J Bradley, Stefan Nicolau, Emma C Frair, Tatyana A. Vetter, Kevin M. Flanigan

المصدر: Neuropathology and applied neurobiology. 48(3)

مصطلحات موضوعية: musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities, Histology, Duchenne muscular dystrophy, Biopsy, Muscle Fibers, Skeletal, Fluorescent Antibody Technique, Immunofluorescence, Pathology and Forensic Medicine, Dystrophin, Tissue markers, Physiology (medical), medicine, Humans, Spectrin, Muscle fibre, Muscle, Skeletal, Sarcolemma, biology, medicine.diagnostic_test, Reproducibility of Results, musculoskeletal system, medicine.disease, Treatment efficacy, Cell biology, Muscular Dystrophy, Duchenne, Neurology, biology.protein, Neurology (clinical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ab91fed38f9ae9d2b05f2c097f2adfc0
https://pubmed.ncbi.nlm.nih.gov/34847621

المؤلفون: Diane M. Dunn, Robert B. Weiss, Megan A. Waldrop, Nicolas Wein, Emma C Frair, Kevin M. Flanigan, Liubov V Gushchina

المصدر: Human Gene Therapy.

مصطلحات موضوعية: 0303 health sciences, biology, Duchenne muscular dystrophy, Computational biology, medicine.disease, Exon skipping, 03 medical and health sciences, Exon, 0302 clinical medicine, 030220 oncology & carcinogenesis, Gene duplication, RNA splicing, Genetics, biology.protein, medicine, Molecular Medicine, splice, Dystrophin, Molecular Biology, Gene, 030304 developmental biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::d9ed0eeb9fb795c5605d09bd17f05f1b
https://doi.org/10.1089/hum.2020.315

المؤلفون: Nicolas, Wein, Diane M, Dunn, Megan A, Waldrop, Liubov V, Gushchina, Emma C, Frair, Robert B, Weiss, Kevin M, Flanigan

المصدر: Human gene therapy. 32(21-22)

مصطلحات موضوعية: Dystrophin, Muscular Dystrophy, Duchenne, Mice, RNA Splicing, RNA, Small Nuclear, Animals, Exons, Genetic Therapy

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid________::d729ce8ecef49001653a5610ea1c3941
https://pubmed.ncbi.nlm.nih.gov/34060935

المؤلفون: Nicolas Wein, Kevin M. Flanigan, Robert B. Weiss, Kim L. McBride, Reid Neinast, Nianyuan Huang, Emma C. Frair, Camila F. Almeida

المصدر: Journal of Visualized Experiments.

مصطلحات موضوعية: General Immunology and Microbiology, General Chemical Engineering, General Neuroscience, General Biochemistry, Genetics and Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::6c2d5e680a21098c79520d00b939f20d
https://doi.org/10.3791/61991-v

المؤلفون: Liubov V, Gushchina, Emma C, Frair, Natalie, Rohan, Adrienne J, Bradley, Tabatha R, Simmons, Hemantkumar D, Chavan, Hsin-Jung, Chou, Michelle, Eggers, Megan A, Waldrop, Nicolas, Wein, Kevin M, Flanigan

المصدر: Human gene therapy. 32(17-18)

مصطلحات موضوعية: Dystrophin, Muscular Dystrophy, Duchenne, Primates, Mice, RNA, Small Nuclear, Animals, Exons

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid________::95fe550ec7788f1301027bef02b71ee5
https://pubmed.ncbi.nlm.nih.gov/33406986

المؤلفون: T. Vetter, Robert B. Weiss, S. Nicolau, Kevin M. Flanigan, Emma C Frair, A. Bradley

المصدر: Neuromuscular Disorders. 31:S79

مصطلحات موضوعية: medicine.medical_specialty, Neurology, business.industry, Pediatrics, Perinatology and Child Health, medicine, Neurology (clinical), Clinical care, Intensive care medicine, business, Genetics (clinical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::b8cb9701e0fd903131c23a1c9f5ca7a9
https://doi.org/10.1016/j.nmd.2021.07.123

المؤلفون: Margaret Beatka, Johan Harris, Brenna R. Powers, Michael W. Lawlor, Tabatha R. Simmons, Tatyana Meyers Vetter, Maryann Kaler, Emma C Frair, Megan A. Waldrop, M. Iammarino, Nico Wein, Hui Meng, Kevin M. Flanigan

المصدر: Neuromuscular Disorders. 30:S166-S167

مصطلحات موضوعية: Presentation, medicine.medical_specialty, Neurology, business.industry, media_common.quotation_subject, General surgery, Pediatrics, Perinatology and Child Health, Medicine, Neurology (clinical), business, Genetics (clinical), media_common

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::26baea78a0e35a45e549635cc39495f3
https://doi.org/10.1016/j.nmd.2020.09.008

المؤلفون: Nicolas Wein, H. Huang, Emma C Frair, K. Grounds, Kevin M. Flanigan, Tabatha R. Simmons, F. Schnell, L. Gushchina, G. Hanson

المصدر: Neuromuscular Disorders. 29:S89

مصطلحات موضوعية: Genetics, Exon, Neurology, Dmd gene, Pediatrics, Perinatology and Child Health, Neurology (clinical), Biology, Genetics (clinical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::eeec0c11a66c37a84586e4ec24448561
https://doi.org/10.1016/j.nmd.2019.06.197