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: Tabatha R. Simmons, Tatyana A. Vetter, Nianyuan Huang, Adeline Vulin-Chaffiol, Nicolas Wein, Kevin M. Flanigan

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 325-340 (2021)

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/S2329050121000541; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/6912488c4ecd404facb01ca8c3e269ec

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: Tatyana A. Vetter, Nicolas Wein, Kevin M. Flanigan, Nianyuan Huang, Tabatha R. Simmons, Adeline Vulin-Chaffiol

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 325-340 (2021)

مصطلحات موضوعية: 0301 basic medicine, musculoskeletal diseases, Duchenne muscular dystrophy, congenital, hereditary, and neonatal diseases and abnormalities, QH426-470, dystrophin, 03 medical and health sciences, Exon, 0302 clinical medicine, Gene duplication, medicine, Genetics, Muscular dystrophy, Molecular Biology, biology, QH573-671, business.industry, medicine.disease, gene therapy, Exon skipping, Open reading frame, Internal ribosome entry site, 030104 developmental biology, Becker muscular dystrophy, 030220 oncology & carcinogenesis, biology.protein, Cancer research, Molecular Medicine, U7snRNA, Dystrophin, business, Cytology, exon skipping

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6ffa2b34b7ee11e1bc422a5d6280fa3d
http://www.sciencedirect.com/science/article/pii/S2329050121000541

المؤلفون: Roxane Alles, Veronica J. Vieland, Robert B. Weiss, Megan A. Waldrop, John Burian, Paul T. Martin, Kevin M. Flanigan, Sang-Cheol Seok, Diane M. Dunn, Melissa Moore-Clingenpeel, Lindsay N. Alfano, Tabatha R. Simmons

مصطلحات موضوعية: musculoskeletal diseases, Candidate gene, Duchenne muscular dystrophy, Locus (genetics), Genome-wide association study, Biology, medicine.disease, Bioinformatics, Sample size determination, Mutation (genetic algorithm), Genetics, medicine, biology.protein, Muscular dystrophy, Dystrophin, Genetics (clinical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::09b1f6360f4a63fecf3342e07518ca40
https://doi.org/10.1101/2021.11.03.21265887

المؤلفون: John Burian, Lindsay N. Alfano, Tabatha R. Simmons, Roxane Alles, Robert B. Weiss, Melissa Moore-Clingenpeel, Diane M. Dunn, Sang-Cheol Seok, Kevin M. Flanigan, Veronica J. Vieland, Paul T. Martin, Megan A. Waldrop

مصطلحات موضوعية: musculoskeletal diseases, Mutation, Candidate gene, Duchenne muscular dystrophy, Single-nucleotide polymorphism, Genome-wide association study, Computational biology, Biology, medicine.disease_cause, medicine.disease, Genome, medicine, Muscular dystrophy, Gene

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::1bf8e8ccebe283ed9b7285e320376e61
https://doi.org/10.1101/2021.11.03.21265899

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: 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: Tabatha R, Simmons, Tatyana A, Vetter, Nianyuan, Huang, Adeline, Vulin-Chaffiol, Nicolas, Wein, Kevin M, Flanigan

المصدر: Molecular Therapy. Methods & Clinical Development

مصطلحات موضوعية: musculoskeletal diseases, Duchenne muscular dystrophy, congenital, hereditary, and neonatal diseases and abnormalities, Becker muscular dystrophy, Original Article, U7snRNA, gene therapy, dystrophin, exon skipping

URL الوصول: https://explore.openaire.eu/search/publication?articleId=pmid________::bfdaed04454052406d7816637fbc62cc
https://pubmed.ncbi.nlm.nih.gov/33898631

المؤلفون: Tabatha R Simmons, Judy F Flax, Marco A Azaro, Jared E Hayter, Laura M Justice, Stephen A Petrill, Anne S Bassett, Paula Tallal, Linda M Brzustowicz, Christopher W Bartlett

المساهمون: The Pennsylvania State University CiteSeerX Archives

المصدر: http://cmbn.rutgers.edu/download/student-publications/Increasing-Genotype-Phenotype-Model-Determinism-Application-to-Bivariate-ReadingLanguage-Traits-and-Epistatic-Interactions-in-Language-Impaired-Families-.pdf.

وصف الملف: application/pdf

Relation: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.1072.3147; http://cmbn.rutgers.edu/download/student-publications/Increasing-Genotype-Phenotype-Model-Determinism-Application-to-Bivariate-ReadingLanguage-Traits-and-Epistatic-Interactions-in-Language-Impaired-Families-.pdf

الاتاحة: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.1072.3147
http://cmbn.rutgers.edu/download/student-publications/Increasing-Genotype-Phenotype-Model-Determinism-Application-to-Bivariate-ReadingLanguage-Traits-and-Epistatic-Interactions-in-Language-Impaired-Families-.pdf

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, D. Li, M. Spencer, C. Young, D. Lesman, C. Gaffney, Y. Rodriguez, D. Rajakumar, Kevin M. Flanigan, R. Rafferty, Nianyuan Huang, Tabatha R. Simmons

المصدر: Neuromuscular Disorders. 30:S107

مصطلحات موضوعية: Neurology, Pediatrics, Perinatology and Child Health, Neurology (clinical), Genetics (clinical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::f9115f0c55d42664d7b559c9c5d8143f
https://doi.org/10.1016/j.nmd.2020.08.208

المؤلفون: María José de Castro, Krista Cope, María L. Couce, MT Oreiro, Kevin M. Flanigan, Louise Jaensch, Maria Fuller, Tabatha R. Simmons, Nicholas J.C. Smith, Juan Ruiz, Kim L. McBride, Kristen V. Truxal

المصدر: Molecular Genetics and Metabolism. 129:S56-S57

مصطلحات موضوعية: medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Biochemistry, Sanfilippo syndrome type a, Gastroenterology, Clinical trial, Endocrinology, ABO blood group system, Internal medicine, Interim, Genetics, medicine, business, Molecular Biology, Mucopolysaccharidosis Type IIIA

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::61ca137efc3d5ff3eb8abf382e976d01
https://doi.org/10.1016/j.ymgme.2019.11.129

المؤلفون: Nicolas Wein, Andrew R. Findlay, Yuuki Kaminoh, Kevin M. Flanigan, Tabatha R. Simmons, Adeline Vulin, A. Rutherford, Jacqueline A. Yurkoski

المصدر: Neuromuscular Disorders. 25:827-834

مصطلحات موضوعية: Male, musculoskeletal diseases, congenital, hereditary, and neonatal diseases and abnormalities, Duchenne muscular dystrophy, Mice, Transgenic, Biology, Exon, Gene Duplication, Gene duplication, medicine, Animals, Gene Knock-In Techniques, RNA, Messenger, Muscle, Skeletal, Creatine Kinase, Genetics (clinical), Body Weight, Intron, Skeletal muscle, Exons, medicine.disease, Phenotype, Molecular biology, Exon skipping, Muscular Dystrophy, Duchenne, Disease Models, Animal, medicine.anatomical_structure, Neurology, Pediatrics, Perinatology and Child Health, Mice, Inbred mdx, biology.protein, Neurology (clinical), Dystrophin

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::181a83c4cdbf4be8b7bd9301eefcb0e8
https://doi.org/10.1016/j.nmd.2015.08.005

المؤلفون: Kim L. McBride, Kristen V. Truxal, Federica Rinaldi, MT Oreiro, María José de Castro, Tabatha R. Simmons, María L. Couce, Maria Fuller, Kevin M. Flanigan, Juan Ruiz

المصدر: Molecular Genetics and Metabolism. 129:S107

مصطلحات موضوعية: medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Safety tolerability, medicine.disease, Biochemistry, Gastroenterology, Mucopolysaccharidosis type IIIB, Clinical trial, Endocrinology, ABO blood group system, Internal medicine, Genetics, medicine, business, Molecular Biology, Sanfilippo syndrome

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::5f4e5d57ebe6eaa1b0cbfcbea1e9d1a0
https://doi.org/10.1016/j.ymgme.2019.11.274

المؤلفون: Lindsay N. Alfano, Tabatha R. Simmons, John P. Cheatham, M. Iammarino, Rui Xu, Sharon L. Cheatham, L. Chicoine, Federica Rinaldi, Paul T. Martin, Linda Lowes, Deborah A. Zygmunt, Kevin M. Flanigan, Megan A. Waldrop, N. Miller

المصدر: Neuromuscular Disorders. 28:S68-S69

مصطلحات موضوعية: Oncology, medicine.medical_specialty, business.industry, Duchenne muscular dystrophy, Gene transfer, medicine.disease, Clinical trial, Phase i ii, Neurology, Internal medicine, Pediatrics, Perinatology and Child Health, Medicine, Neurology (clinical), business, Genetics (clinical)

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::fdc04c636c4e83cd1e22350975c8f749
https://doi.org/10.1016/j.nmd.2018.06.159

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

المؤلفون: Eric K. Johnson, James M. Ervasti, Dana M. Strandjord, Nicolas Wein, William C. Ray, Andrew R. Findlay, Kevin M. Flanigan, Michael T. Howard, Yuuki Kaminoh, Adeline Vulin, Laura E. Taylor, Tabatha R. Simmons, Federica Montanaro, Baijayanta Maiti

المصدر: Human Mutation. 35:257-264

مصطلحات موضوعية: musculoskeletal diseases, Protein Folding, congenital, hereditary, and neonatal diseases and abnormalities, Duchenne muscular dystrophy, Mutation, Missense, Mice, Transgenic, medicine.disease_cause, Article, Dystrophin, WW domain, Mice, Glycoprotein complex, Utrophin, Genetics, medicine, Animals, Humans, Missense mutation, Cysteine, Dystroglycans, Genetics (clinical), Aspartic Acid, Mutation, biology, Protein Stability, Genetic Variation, Zinc Fingers, musculoskeletal system, medicine.disease, Actins, Transmembrane protein, Muscular Dystrophy, Duchenne, biology.protein

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b4ef972ba1cc0aef92bfd2a9050991e4
https://doi.org/10.1002/humu.22479