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1دورية أكاديمية
المؤلفون: Laura J. Smith, Lindsay A. Schulman, Samantha Smith, Laura Van Lieshout, Carmen M. Barnes, Liana Behmoiras, Meghan Scarpitti, Monicah Kivaa, Khanh L. Duong, Ludo O. Benard, Jeff L. Ellsworth, Nancy Avila, Deiby Faulkner, April Hayes, Jason Lotterhand, Jose Israel Rivas, Arnold V. Sengooba, Alec Tzianabos, Albert B. Seymour, Omar L. Francone
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 224-238 (2022)
مصطلحات موضوعية: gene therapy, adeno-associated virus, AAV, tropism/biodistribution, glycan binding, structure-function relationship, Genetics, QH426-470, Cytology, QH573-671
وصف الملف: electronic resource
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2دورية أكاديمية
المؤلفون: Seemin S. Ahmed, Hillard Rubin, Minglun Wang, Deiby Faulkner, Arnold Sengooba, Serena N. Dollive, Nancy Avila, Jeff L. Ellsworth, Diana Lamppu, Maria Lobikin, Jason Lotterhand, Laura Adamson-Small, Teresa Wright, Albert Seymour, Omar L. Francone
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 568-580 (2020)
وصف الملف: electronic resource
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3
المؤلفون: Minglun Wang, Hillard Rubin, Jason Lotterhand, Omar L. Francone, Maria Lobikin, Seemin Seher Ahmed, Albert B. Seymour, Diana Lamppu, Laura Adamson-Small, Serena Nicole Dollive, Jeff L. Ellsworth, Teresa Leah Wright, Nancy Avila, Arnold Sengooba, Deiby Faulkner
المصدر: Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 568-580 (2020)مصطلحات موضوعية: 0301 basic medicine, lcsh:QH426-470, Phenylalanine hydroxylase, Genetic enhancement, Metabolite, Transgene, Phenylalanine, Pharmacology, Article, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Genetics, medicine, lcsh:QH573-671, Tyrosine, Molecular Biology, chemistry.chemical_classification, biology, lcsh:Cytology, business.industry, medicine.disease, lcsh:Genetics, 030104 developmental biology, Enzyme, chemistry, Inborn error of metabolism, 030220 oncology & carcinogenesis, biology.protein, Molecular Medicine, business
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4
المؤلفون: Victor Zhivivh, Nancy Avila, Jingyan Zhao, Omar L. Francone, Jacinthe Gingras, Kruti Patel, Albert B. Seymour, Monicah Kivaa, Liana Behmoiras, Tania A. Seabrook, Laura Smith, Steve Woodcock, Alec Tzianabos, Arnold Sengooba, Lindsay Schulman, Deiby Faulkner, Serena Nicole Dollive, Jennifer Newman, Yvonne White, Sean Smith
المصدر: Molecular Genetics and Metabolism. 132:S147
مصطلحات موضوعية: Endocrinology, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Immunology, Genetics, medicine, Hunter syndrome, Mucopolysaccharidosis type II, medicine.disease, business, Molecular Biology, Biochemistry
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5HMI-203: Investigational gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome
المؤلفون: Albert B. Seymour, Monicah Kivaa, Liana Behmoiras, Laura Smith, Lindsay Schulman, Sean Smith, Serena Nicole Dollive, Kruti Patel, Deiby Faulkner, Nancy Avila, Victor Zhivich, Omar L. Francone, Yvonne White, Alec Tzianabos, Arnold Sengooba, Tania A. Seabrook, Jenn Newman, Jacinthe Gingras, Steve Woodcock, Jingyan Zhao
المصدر: Molecular Genetics and Metabolism. 132:S82
مصطلحات موضوعية: Oncology, medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Hunter syndrome, INVESTIGATIONAL GENE THERAPY, medicine.disease, Biochemistry, Endocrinology, Internal medicine, Genetics, medicine, Mucopolysaccharidosis type II, business, Molecular Biology
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6
المؤلفون: Hillard Rubin, Diana Lamppu, Omar L. Francone, Deiby Faulkner, Serena Nicole Dollive, Seemin Seher Ahmed, Arnold Sengooba, Jeff L. Ellsworth, Albert B. Seymour, Teresa Leah Wright
المصدر: Molecular Genetics and Metabolism. 126:S56
مصطلحات موضوعية: Intravenous dose, Endocrinology, Phenylalanine hydroxylase, biology, Chemistry, Endocrinology, Diabetes and Metabolism, Transgene, Genetics, biology.protein, Vector (molecular biology), Pharmacology, Molecular Biology, Biochemistry