Zobrazeno 1 - 10
of 1 077
pro vyhledávání: '"iduronate"'
Autor:
Al-Hertani W; Division of Genetics and Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Pathak RR; Takeda Pharmaceuticals USA, Inc., Lexington, MA 02421, USA., Evuarherhe O; Oxford PharmaGenesis, Oxford OX13 5QJ, UK., Carter G; Oxford PharmaGenesis, Oxford OX13 5QJ, UK., Schaeffer-Koziol CR; Takeda Pharmaceuticals USA, Inc., Lexington, MA 02421, USA., Whiteman DAH; Takeda Development Center Americas, Inc., Lexington, MA 02421, USA., Wright E; Takeda Development Center Americas, Inc., Lexington, MA 02421, USA.
Publikováno v:
International journal of molecular sciences [Int J Mol Sci] 2024 Aug 06; Vol. 25 (16). Date of Electronic Publication: 2024 Aug 06.
Autor:
Das S; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA 02115, USA., Rruga F; Division of Hematology, Oncology and Stem Cell Transplantation, Department of Women and Child's Health, University of Padua, 35128 Padua, Italy., Montepeloso A; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA 02115, USA., Dimartino A; Division of Hematology, Oncology and Stem Cell Transplantation, Department of Women and Child's Health, University of Padua, 35128 Padua, Italy., Spadini S; Division of Hematology, Oncology and Stem Cell Transplantation, Department of Women and Child's Health, University of Padua, 35128 Padua, Italy., Corre G; Genethon, 91000 Evry, France., Patel J; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA 02115, USA., Cavalca E; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA 02115, USA., Ferro F; Gene Therapy Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Boston, MA 02115, USA., Gatti A; AGC Biologics, 20091 Milan, Italy., Milazzo R; Altheia Science s.r.l., 20122 Milan, Italy., Galy A; Genethon, 91000 Evry, France., Politi LS; Humanitas University and IRCCS Humanitas Research Hospital, 20090 Pieve Emanuele (MI), Italy., Rizzardi GP; Altheia Science s.r.l., 20122 Milan, Italy., Vallanti G; AGC Biologics, 20091 Milan, Italy., Poletti V; Division of Hematology, Oncology and Stem Cell Transplantation, Department of Women and Child's Health, University of Padua, 35128 Padua, Italy., Biffi A; Division of Hematology, Oncology and Stem Cell Transplantation, Department of Women and Child's Health, University of Padua, 35128 Padua, Italy. Electronic address: alessandra.biffi@unipd.it.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Mar 06; Vol. 32 (3), pp. 619-636. Date of Electronic Publication: 2024 Feb 03.
Autor:
Mandolfo O; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom., Liao A; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom., Singh E; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom., O'leary C; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom., Holley RJ; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom., Bigger BW; Stem Cell and Neurotherapies, Division of Cell Matrix Biology and Regenerative Medicine, Faculty of Biology, Medicine and Health, University of Manchester, Manchester, United Kingdom.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2024 Apr; Vol. 35 (7-8), pp. 243-255. Date of Electronic Publication: 2023 Aug 30.
Autor:
Delgado CA; Programa de Pós-Graduação em Ciências Biológicas: Bioquímica, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.; Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil., Poletto E; Programa de Pós-Graduação em Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.; Centro de Terapia Gênica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil., Vera LNP; Programa de Pós-Graduação em Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.; Centro de Terapia Gênica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil., Jacques CED; Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil., Vianna P; Departamento de Genética, Instituto de Biociências, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil., Reinhardt LS; Priority Research, University of Newcastle, Newcastle, Australia., Baldo G; Programa de Pós-Graduação em Biologia Molecular, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.; Centro de Terapia Gênica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.; Departamento de Fisiologia, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil., Vargas CR; Programa de Pós-Graduação em Ciências Biológicas: Bioquímica, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil.; Serviço de Genética Médica, Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil.
Publikováno v:
Cell biochemistry and function [Cell Biochem Funct] 2024 Mar; Vol. 42 (2), pp. e3932.
Autor:
Manzoli R; Department of Molecular Medicine, University of Padova, 35121, Padova, Italy. rosa.manzoli@unipd.it.; Department of Biology, University of Padova, 35121, Padova, Italy. rosa.manzoli@unipd.it., Badenetti L; Department of Molecular Medicine, University of Padova, 35121, Padova, Italy.; Department of Women's and Children's Health, University of Padova, 35128, Padova, Italy.; Istituto di Ricerca Pediatrica 'Città Della Speranza', 35127, Padova, Italy., Bruzzone M; Department of Biomedical Sciences, University of Padova, 35121, Padova, Italy.; Padua Neuroscience Center - PNC, University of Padova, 35129, Padova, Italy., Macario MC; Department of Molecular Medicine, University of Padova, 35121, Padova, Italy.; Department of Biology, University of Padova, 35121, Padova, Italy., Rubin M; Department of Molecular Medicine, University of Padova, 35121, Padova, Italy., Dal Maschio M; Department of Biomedical Sciences, University of Padova, 35121, Padova, Italy.; Padua Neuroscience Center - PNC, University of Padova, 35129, Padova, Italy., Roveri A; Department of Molecular Medicine, University of Padova, 35121, Padova, Italy., Moro E; Department of Molecular Medicine, University of Padova, 35121, Padova, Italy. enrico.moro.1@unipd.it.
Publikováno v:
Cell death & disease [Cell Death Dis] 2024 Apr 16; Vol. 15 (4), pp. 269. Date of Electronic Publication: 2024 Apr 16.
Autor:
Yee KS; Takeda Development Center Americas, Inc., Cambridge, MA, USA.; Present affiliation: Alexion Pharmaceuticals, Inc., AstraZeneca Rare Disease, Boston, MA, USA., Alexanderian D; Takeda Development Center Americas, Inc., Lexington, MA, USA.; Present affiliation: Merck, Boston, MA, USA., Martin S; RTI Health Solutions, Ann Arbor, MI, USA., Olayinka-Amao B; RTI Health Solutions, Research Triangle Park, NC, USA., Whiteman DAH; Takeda Development Center Americas, Inc., Lexington, MA, USA. dahw.md@gmail.com.
Publikováno v:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Apr 12; Vol. 19 (1), pp. 158. Date of Electronic Publication: 2024 Apr 12.
Autor:
Catalano F; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Vlaar EC; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Dammou Z; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Katsavelis D; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Huizer TF; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Zundo G; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Hoogeveen-Westerveld M; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Oussoren E; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., van den Hout HJMP; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Schaaf G; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Pike-Overzet K; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands., Staal FJT; Department of Immunology, Leiden University Medical Center, Leiden, The Netherlands.; Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands., van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Pijnappel WWMP; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam, The Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2024 Apr; Vol. 35 (7-8), pp. 256-268. Date of Electronic Publication: 2024 Feb 02.
Autor:
Wood SR; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Chaudrhy A; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Ellison S; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Searle R; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Burgod C; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Tehseen G; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Forte G; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., O'Leary C; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Gleitz H; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom.; Department of Developmental Biology, Erasmus University, Rotterdam, the Netherlands., Liao A; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom.; Department of Infection, Immunity and Inflammation,University College London, London, United Kingdom., Cook J; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Holley R; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom., Bigger BW; Division of Cell Matrix Biology and Regenerative Medicine, University of Manchester, Manchester, United Kingdom.
Publikováno v:
Human gene therapy [Hum Gene Ther] 2024 Apr; Vol. 35 (7-8), pp. 232-242. Date of Electronic Publication: 2023 Jul 07.
Autor:
Yee KS; Takeda Development Center Americas, Inc., Cambridge, MA, USA. kyee22_@hotmail.com., Lewis S; RTI Health Solutions, Research Triangle Park, Raleigh, NC, USA., Evans E; RTI Health Solutions, Research Triangle Park, Raleigh, NC, USA., Romano C; RTI Health Solutions, Research Triangle Park, Raleigh, NC, USA., Alexanderian D; Takeda Development Center Americas, Inc., Lexington, MA, USA.
Publikováno v:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Mar 10; Vol. 19 (1), pp. 110. Date of Electronic Publication: 2024 Mar 10.
Autor:
Nakamura K; Department of Pediatrics, Faculty of Life Science, Kumamoto University, 860-0862, Kumamoto, Japan., Sakai N; Child Healthcare and Genetic Science Laboratory, Division of Health Sciences, Osaka University Graduate School of Medicine, 565-0871, Osaka, Japan., Hossain MA; JCR Pharmaceuticals, 11-18 Kusunoki-cho, 659-0015, Ashiya city, Hyogo, Japan. arif@jp.jcrpharm.com., Eisengart JB; Department of Pediatrics, University of Minnesota, 55455, Minneapolis, MN, USA., Yamamoto T; JCR Pharmaceuticals, 11-18 Kusunoki-cho, 659-0015, Ashiya city, Hyogo, Japan., Tanizawa K; JCR Pharmaceuticals, 11-18 Kusunoki-cho, 659-0015, Ashiya city, Hyogo, Japan., So S; JCR Pharmaceuticals, 11-18 Kusunoki-cho, 659-0015, Ashiya city, Hyogo, Japan., Schmidt M; JCR Pharmaceuticals, 11-18 Kusunoki-cho, 659-0015, Ashiya city, Hyogo, Japan., Sato Y; JCR Pharmaceuticals, 11-18 Kusunoki-cho, 659-0015, Ashiya city, Hyogo, Japan.
Publikováno v:
Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Mar 07; Vol. 19 (1), pp. 104. Date of Electronic Publication: 2024 Mar 07.