نتائج البحث - "Nicola Brunetti-Pierri"

1

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism

المؤلفون: Nicola Brunetti-Pierri, Alberto Auricchio, Rita Ferla, Virginia Maria Ginocchio

المساهمون: Ginocchio, V. M., Ferla, R., Auricchio, A., Brunetti-Pierri, N.

المصدر: Human gene therapy. 30(10)

مصطلحات موضوعية: Genetic enhancement, Genetic Vectors, inborn errors of metabolism, Gene delivery, Bioinformatics, medicine.disease_cause, Virus, 03 medical and health sciences, Liver disease, adeno-associated virus vector, 0302 clinical medicine, Genome editing, Genetics, Medicine, Animals, Humans, Molecular Biology, Adeno-associated virus, 030304 developmental biology, Gene Editing, 0303 health sciences, Newborn screening, Clinical Trials as Topic, business.industry, Liver Diseases, Lentivirus, Gene Transfer Techniques, AAV, Genetic Therapy, Dependovirus, medicine.disease, gene therapy, Clinical trial, Liver, 030220 oncology & carcinogenesis, Hepatocytes, Molecular Medicine, business, Metabolism, Inborn Errors

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a0c32744e13353462f9455aeff7facd9Test
https://pubmed.ncbi.nlm.nih.gov/31517544Test

2

Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome

المؤلفون: Sem J. Aronson, Angelo Di Giorgio, Ulrich Baumann, Virginie Delahais, Lorenzo D'Antiga, Fanny Collaud, Aurélie Hubert, Géraldine Honnet, Federico Mingozzi, Nicola Brunetti-Pierri, Philippe Labrune, Norman Junge, Robert J. de Knegt, Ulrich Beuers, Virginia Maria Ginocchio, Piter J. Bosma, Philippe Veron

المساهمون: 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, AP-HP - Hôpital Antoine Béclère [Clamart], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Gastroenterology and Hepatology, Erasmus University Medical Center [Rotterdam] (Erasmus MC), Università degli studi di Napoli Federico II, University of Amsterdam [Amsterdam] (UvA), Graduate School, Tytgat Institute for Liver and Intestinal Research, AGEM - Digestive immunity, AGEM - Endocrinology, metabolism and nutrition, AGEM - Inborn errors of metabolism, École Pratique des Hautes Études (EPHE), University of Naples Federico II = Università degli studi di Napoli Federico II, Gastroenterology & Hepatology, Aronson, S. J., Veron, P., Collaud, F., Hubert, A., Delahais, V., Honnet, G., De Knegt, R. J., Junge, N., Baumann, U., Di Giorgio, A., D'Antiga, L., Ginocchio, virginia maria, Brunetti-Pierri, N., Labrune, P., Beuers, U., Bosma, P. J., Mingozzi, F., Laboratoire Traitement du Signal et de l'Image (LTSI), Université de Rennes 1 (UR1), Université de Rennes (UNIV-RENNES)-Université de Rennes (UNIV-RENNES)-Institut National de la Santé et de la Recherche Médicale (INSERM), École pratique des hautes études (EPHE)-Université d'Évry-Val-d'Essonne (UEVE)-GENETHON 3-Institut National de la Santé et de la Recherche Médicale (INSERM), COMUE Université Côte d'Azur (2015 - 2019) (COMUE UCA)

المصدر: Human Gene Therapy
Human Gene Therapy, Mary Ann Liebert, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human gene therapy, 30(10), 1297-1305. Mary Ann Liebert Inc.
Human Gene Therapy, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩
Human Gene Therapy, 30(10), 1297-1305. Mary Ann Liebert Inc.
Hum Gene Ther
Hum Gene Ther, 2019, 30, pp.1297-1305. ⟨10.1089/hum.2019.143⟩

مصطلحات موضوعية: Serotype, Male, anti-AAV neutralizing antibodie, Crigler–Najjar syndrome, [SDV]Life Sciences [q-bio], Genetic enhancement, anti-AAV neutralizing antibodies, Gene Expression, medicine.disease_cause, Antibodies, Viral, [SDV.IMM.II]Life Sciences [q-bio]/Immunology/Innate immunity, Mice, 0302 clinical medicine, Glucuronosyltransferase, Child, Adeno-associated virus, Research Articles, Crigler-Najjar Syndrome, 0303 health sciences, biology, [SDV.MHEP.EM]Life Sciences [q-bio]/Human health and pathology/Endocrinology and metabolism, Dependovirus, 3. Good health, Titer, unconjugated hyperbilirubinemia, [SDV.IMM.IA]Life Sciences [q-bio]/Immunology/Adaptive immunology, Liver, 030220 oncology & carcinogenesis, Child, Preschool, Phenobarbital, Molecular Medicine, Female, Antibody, AAV gene therapy, Plasmids, Adult, Adolescent, pre-existing immunity, Transfection, Virus, 03 medical and health sciences, Capsid, Immunity, Genetics, medicine, Animals, Humans, Molecular Biology, 030304 developmental biology, business.industry, Immunization, Passive, Bilirubin, Genetic Therapy, Phototherapy, medicine.disease, Antibodies, Neutralizing, Immunity, Innate, Mice, Inbred C57BL, HEK293 Cells, Immunology, biology.protein, UGT1A1, business, Excitatory Amino Acid Antagonists

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5c1a2d9c9f9b11cded8a89710f8f8580Test
https://pubmed.ncbi.nlm.nih.gov/31502485Test

3

Gene Therapy for Inherited Diseases of Liver Metabolism

المؤلفون: Pasquale Piccolo, Nicola Brunetti-Pierri

المساهمون: Piccolo, P, BRUNETTI PIERRI, Nicola

المصدر: Human Gene Therapy. 26:186-192

مصطلحات موضوعية: Genetics, Clinical Trials as Topic, Liver Diseases, Genetic enhancement, Genetic Vectors, Genetic Therapy, Biology, Bioinformatics, Genetic therapy, Liver metabolism, Metabolic Diseases, Animals, Humans, Molecular Medicine, Molecular Biology

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cdd4c2c4d7fd71d1e26e635209e58edcTest
https://doi.org/10.1089/hum.2015.029Test

4

Prevalence of Anti–Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates for a Gene Therapy Trial

المؤلفون: Giancarlo Parenti, Karen Kozarsky, John J. Hopwood, Rita Ferla, Generoso Andria, Alberto Auricchio, Ans T. van der Ploeg, Rossella Parini, Marco Savarese, Maurizio Scarpa, Hatice Serap Sivri, Vincenzo Nigro, Pamela Claudiani, Nicola Brunetti-Pierri, Maria Alice Donati, Giovanni Sorge, Simona Fecarotta

المساهمون: Ferla, R, Claudiani, P, Savarese, M, Kozarsky, K., Parini, R, Scarp, a M, Donati, Ma, Sorge, G, Hopwood, Jj, Parenti, Giancarlo, Fecarotta, S, Nigro, V, Sivri, H, Van Der Ploeg, A, Andria, Generoso, BRUNETTI PIERRI, Nicola, Auricchio, Alberto, Çocuk Sağlığı ve Hastalıkları, Ferla, Rita, Claudiani, Pamela, Savarese, Marco, Kozarsky, Karen, Parini, Rossella, Scarpa, Maurizio, Donati, Maria Alice, Sorge, Giovanni, Hopwood, John J., Fecarotta, Simona, Nigro, Vincenzo, Sivri, Hatice Serap, Van Der Ploeg, An, Brunetti Pierri, Nicola, Pediatrics

المصدر: Human Gene Therapy
Human Gene Therapy, 26(3), 145-152. Mary Ann Liebert Inc.

مصطلحات موضوعية: Arylsulfatase B, Turkey, N-Acetylgalactosamine-4-Sulfatase, Genetic enhancement, DNA Mutational Analysis, medicine.disease_cause, Cohort Studies, 0302 clinical medicine, Medicine, Neutralizing, Adeno-associated virus, Research Articles, Netherlands, 0303 health sciences, Mucopolysaccharidosis VI, biology, Medicine (all), Dependovirus, Dependoviru, 3. Good health, Italy, 030220 oncology & carcinogenesis, Cross Reaction, Molecular Medicine, Antibody, Human, Cross Reactions, Antibodies, Neutralizing, Genetic Therapy, Humans, Mutation, Patient Selection, Molecular Biology, Genetics, Antibodies, Virus, Frameshift mutation, DNA Mutational Analysi, 03 medical and health sciences, Netherland, 030304 developmental biology, business.industry, Virology, Immunology, biology.protein, Cohort Studie, business