Zobrazeno 1 - 10
of 304
pro vyhledávání: '"van Til NP"'
Autor:
Yoon JK; AVROBIO, Inc., Cambridge, MA 02139, USA., Schindler JW; AVROBIO, Inc., Cambridge, MA 02139, USA., Loperfido M; AVROBIO, Inc., Cambridge, MA 02139, USA., Baricordi C; AVROBIO, Inc., Cambridge, MA 02139, USA., DeAndrade MP; AVROBIO, Inc., Cambridge, MA 02139, USA., Jacobs ME; AVROBIO, Inc., Cambridge, MA 02139, USA., Treleaven C; AVROBIO, Inc., Cambridge, MA 02139, USA., Plasschaert RN; AVROBIO, Inc., Cambridge, MA 02139, USA., Yan A; AVROBIO, Inc., Cambridge, MA 02139, USA., Barese CN; AVROBIO, Inc., Cambridge, MA 02139, USA., Dogan Y; AVROBIO, Inc., Cambridge, MA 02139, USA., Chen VP; AVROBIO, Inc., Cambridge, MA 02139, USA., Fiorini C; AVROBIO, Inc., Cambridge, MA 02139, USA., Hull F; AVROBIO, Inc., Cambridge, MA 02139, USA., Barbarossa L; AVROBIO, Inc., Cambridge, MA 02139, USA., Unnisa Z; AVROBIO, Inc., Cambridge, MA 02139, USA., Ivanov D; AVROBIO, Inc., Cambridge, MA 02139, USA., Kutner RH; AVROBIO, Inc., Cambridge, MA 02139, USA., Guda S; AVROBIO, Inc., Cambridge, MA 02139, USA., Oborski C; AVROBIO, Inc., Cambridge, MA 02139, USA., Maiwald T; AVROBIO, Inc., Cambridge, MA 02139, USA., Michaud V; Lady Davis Institute for Medical Research, Jewish General Hospital, McGill University, Montreal, Quebec H3T 1E2, Canada., Rothe M; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Straße 1, 30625 Hannover, Germany., Schambach A; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Straße 1, 30625 Hannover, Germany; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Pfeifer R; AVROBIO, Inc., Cambridge, MA 02139, USA., Mason C; AVROBIO, Inc., Cambridge, MA 02139, USA; Advanced Centre for Biochemical Engineering, University College London, London WC1E 6AE, UK., Biasco L; AVROBIO, Inc., Cambridge, MA 02139, USA; Zayed Centre for Research, University College London, London WC1N 1DZ, UK., van Til NP; AVROBIO, Inc., Cambridge, MA 02139, USA; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Center, VU University, and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1081 HV, Amsterdam, the Netherlands; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, 1081 HV, Amsterdam, the Netherlands. Electronic address: n.p.vantil@amsterdamumc.nl.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 Sep 17. Date of Electronic Publication: 2024 Sep 17.
Autor:
Plug BC; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands., Revers IM; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands., Breur M; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands., González GM; Department of Anatomy and Neurosciences, MS Center Amsterdam, Amsterdam University Medical Centre, VU University, Amsterdam Neuroscience, De Boelelaan 1108, Amsterdam, 1081 HZ, The Netherlands., Timmerman JA; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam Neuroscience, De Boelelaan 1085, Amsterdam, 1081 HV, The Netherlands., Meijns NRC; Department of Anatomy and Neurosciences, MS Center Amsterdam, Amsterdam University Medical Centre, VU University, Amsterdam Neuroscience, De Boelelaan 1108, Amsterdam, 1081 HZ, The Netherlands., Hamberg D; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands., Wagendorp J; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands., Nutma E; Department of Pathology, Amsterdam Neuroscience, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands., Wolf NI; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands., Luchicchi A; Department of Anatomy and Neurosciences, MS Center Amsterdam, Amsterdam University Medical Centre, VU University, Amsterdam Neuroscience, De Boelelaan 1108, Amsterdam, 1081 HZ, The Netherlands., Mansvelder HD; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam Neuroscience, De Boelelaan 1085, Amsterdam, 1081 HV, The Netherlands., van Til NP; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands.; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam Neuroscience, De Boelelaan 1085, Amsterdam, 1081 HV, The Netherlands., van der Knaap MS; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands.; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam Neuroscience, De Boelelaan 1085, Amsterdam, 1081 HV, The Netherlands., Bugiani M; Department of Paediatrics and Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands. m.bugiani@amsterdamumc.nl.; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centre, Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Meibergdreef 9, 1100 DD, Amsterdam, The Netherlands. m.bugiani@amsterdamumc.nl.; Department of Pathology, Amsterdam Neuroscience, Amsterdam University Medical Centre, Meibergdreef 9, Amsterdam, 1100 DD, The Netherlands. m.bugiani@amsterdamumc.nl.
Publikováno v:
Acta neuropathologica communications [Acta Neuropathol Commun] 2024 May 31; Vol. 12 (1), pp. 83. Date of Electronic Publication: 2024 May 31.
Autor:
Böck D; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland., Revers IM; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1105AZ Amsterdam, the Netherlands., Bomhof ASJ; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1105AZ Amsterdam, the Netherlands., Hillen AEJ; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1105AZ Amsterdam, the Netherlands., Boeijink C; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1105AZ Amsterdam, the Netherlands., Kissling L; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland., Egli S; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland., Moreno-Mateos MA; Andalusian Center for Developmental Biology (CABD), Pablo de Olavide University/CSIC/Junta de Andalucía, 41013 Seville, Spain; Department of Molecular Biology and Biochemical Engineering, Pablo de Olavide University, 41013 Seville, Spain., van der Knaap MS; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1105AZ Amsterdam, the Netherlands; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, 1081HV Amsterdam, the Netherlands., van Til NP; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1105AZ Amsterdam, the Netherlands; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, 1081HV Amsterdam, the Netherlands. Electronic address: n.p.vantil@amsterdamumc.nl., Schwank G; Institute of Pharmacology and Toxicology, University of Zurich, 8057 Zurich, Switzerland. Electronic address: schwank@pharma.uzh.ch.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2024 May 01; Vol. 32 (5), pp. 1328-1343. Date of Electronic Publication: 2024 Mar 07.
Autor:
Lo Presti V; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands., Meringa A; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands., Dunnebach E; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., van Velzen A; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., Moreira AV; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., Stam RW; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., Kotecha RS; Department of Clinical Haematology, Oncology, Blood and Marrow Transplantation, Perth Children's Hospital, Perth, Western Australia, Australia.; University of Western Australia, Perth, Western Australia, Australia., Krippner-Heidenreich A; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., Heidenreich OT; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands., Plantinga M; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands., Cornel A; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands., Sebestyen Z; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands., Kuball J; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands., van Til NP; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit Amsterdam and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, Amsterdam, The Netherlands.; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, Amsterdam, The Netherlands., Nierkens S; Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands S.Nierkens-2@prinsesmaximacentrum.nl.; Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
Publikováno v:
Journal for immunotherapy of cancer [J Immunother Cancer] 2024 Apr 05; Vol. 12 (4). Date of Electronic Publication: 2024 Apr 05.
Autor:
Liang Q; Department of Hematology and Research Laboratory of Hematology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, PR China; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Vlaar EC; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Pijnenburg JM; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Rijkers E; Proteomics Center, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Demmers JAA; Proteomics Center, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Vulto AG; Hospital Pharmacy, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van Til NP; Department of Hematology, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Pijnappel WWMP; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands. Electronic address: w.pijnappel@erasmusmc.nl.
Publikováno v:
Journal of proteomics [J Proteomics] 2024 Jan 16; Vol. 291, pp. 105037. Date of Electronic Publication: 2023 Oct 30.
Autor:
Aerts-Kaya F; Department of Stem Cell Sciences, Graduate School of Health Sciences, Center for Stem Cell Research and Development, Hacettepe University, 06100 Ankara, Turkey.; Advanced Technologies Application and Research Center, Hacettepe University, 06800 Ankara, Turkey., van Til NP; Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, Amsterdam Neuroscience, 1081 HV Amsterdam, The Netherlands.; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, Vrije Universiteit Amsterdam, 1081 HV Amsterdam, The Netherlands.
Publikováno v:
Pharmaceutics [Pharmaceutics] 2023 Oct 24; Vol. 15 (11). Date of Electronic Publication: 2023 Oct 24.
Autor:
Bugiani M; Department of Pathology, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands.; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands., Abbink TEM; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.; Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands., Edridge AWD; Laboratory of Experimental Virology, Department of Medical Microbiology and Infection Prevention, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands.; Amsterdam Centre for Global Child Health, Amsterdam University Medical Centers, Amsterdam, The Netherlands., van der Hoek L; Laboratory of Experimental Virology, Department of Medical Microbiology and Infection Prevention, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam, The Netherlands., Hillen AEJ; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.; Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands., van Til NP; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.; Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands., Hu-A-Ng GV; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.; Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands., Breur M; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.; Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands., Aiach K; Lysogene, Neuilly-sur-Seine, France., Drevot P; Lysogene, Neuilly-sur-Seine, France., Hocquemiller M; Lysogene, Neuilly-sur-Seine, France., Laufer R; Lysogene, Neuilly-sur-Seine, France., Wijburg FA; Department of Pediatric Metabolic Diseases, Emma Children's Hospital and Amsterdam Lysosome Center 'Sphinx', Amsterdam University Medical Centers, Academic Medical Center, Amsterdam, The Netherlands., van der Knaap MS; Amsterdam Leukodystrophy Center, Amsterdam University Medical Centers, Amsterdam, The Netherlands.; Department of Child Neurology, Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience, Amsterdam, The Netherlands.; Department of Integrative Neurophysiology, Center for Neurogenomics and Cognitive Research, VU University, Amsterdam, 1081 HV, The Netherlands.
Publikováno v:
Annals of clinical and translational neurology [Ann Clin Transl Neurol] 2023 Jun; Vol. 10 (6), pp. 904-917. Date of Electronic Publication: 2023 May 11.
Autor:
Dogan Y; AVROBIO, Inc., Cambridge, MA 02139, USA., Barese CN; AVROBIO, Inc., Cambridge, MA 02139, USA., Schindler JW; AVROBIO, Inc., Cambridge, MA 02139, USA., Yoon JK; AVROBIO, Inc., Cambridge, MA 02139, USA., Unnisa Z; AVROBIO, Inc., Cambridge, MA 02139, USA., Guda S; AVROBIO, Inc., Cambridge, MA 02139, USA., Jacobs ME; AVROBIO, Inc., Cambridge, MA 02139, USA., Oborski C; AVROBIO, Inc., Cambridge, MA 02139, USA., Maiwald T; AVROBIO, Inc., Cambridge, MA 02139, USA., Clarke DL; AVROBIO, Inc., Cambridge, MA 02139, USA., Schambach A; Institute of Experimental Hematology, Hannover Medical School, Carl-Neuberg-Straße 1, 30625 Hannover, Germany.; Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA., Pfeifer R; AVROBIO, Inc., Cambridge, MA 02139, USA., Harper C; AVROBIO, Inc., Cambridge, MA 02139, USA., Mason C; AVROBIO, Inc., Cambridge, MA 02139, USA.; Advanced Centre for Biochemical Engineering, University College London, London WC1E 6AE, UK., van Til NP; AVROBIO, Inc., Cambridge, MA 02139, USA.; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, VU University, and Amsterdam Neuroscience, Cellular & Molecular Mechanisms, 1081 HV Amsterdam, the Netherlands.
Publikováno v:
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2022 Nov 03; Vol. 27, pp. 464-487. Date of Electronic Publication: 2022 Nov 03 (Print Publication: 2022).
Autor:
Plasschaert RN; AVROBIO, Inc, Cambridge, MA 02139, USA., DeAndrade MP; AVROBIO, Inc, Cambridge, MA 02139, USA., Hull F; AVROBIO, Inc, Cambridge, MA 02139, USA., Nguyen Q; AVROBIO, Inc, Cambridge, MA 02139, USA., Peterson T; AVROBIO, Inc, Cambridge, MA 02139, USA., Yan A; AVROBIO, Inc, Cambridge, MA 02139, USA., Loperfido M; AVROBIO, Inc, Cambridge, MA 02139, USA., Baricordi C; AVROBIO, Inc, Cambridge, MA 02139, USA., Barbarossa L; AVROBIO, Inc, Cambridge, MA 02139, USA., Yoon JK; AVROBIO, Inc, Cambridge, MA 02139, USA., Dogan Y; AVROBIO, Inc, Cambridge, MA 02139, USA., Unnisa Z; AVROBIO, Inc, Cambridge, MA 02139, USA., Schindler JW; AVROBIO, Inc, Cambridge, MA 02139, USA., van Til NP; AVROBIO, Inc, Cambridge, MA 02139, USA; Department of Child Neurology, Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers, VU University, and Amsterdam Neuroscience, Amsterdam, the Netherlands., Biasco L; AVROBIO, Inc, Cambridge, MA 02139, USA; Great Ormond Street Institute of Child Health, University College London, London, UK., Mason C; AVROBIO, Inc, Cambridge, MA 02139, USA; Advanced Centre for Biochemical Engineering, University College London, London, UK. Electronic address: chris.mason@ucl.ac.uk.
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2022 Oct 05; Vol. 30 (10), pp. 3209-3225. Date of Electronic Publication: 2022 May 25.
Autor:
Liang Q; Department of Hematology and Research Laboratory of Hematology, West China Hospital, Sichuan University, Chengdu, Sichuan 610041, P. R. China.; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Catalano F; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Vlaar EC; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Pijnenburg JM; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Stok M; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Hematology, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van Helsdingen Y; Department of Hematology, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Vulto AG; Hospital Pharmacy, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van der Ploeg AT; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., van Til NP; Department of Hematology, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands., Pijnappel WWMP; Department of Clinical Genetics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Department of Pediatrics, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.; Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Rotterdam 3015GE, the Netherlands.
Publikováno v:
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2022 Sep 24; Vol. 27, pp. 109-130. Date of Electronic Publication: 2022 Sep 24 (Print Publication: 2022).