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Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease.

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Title: Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease.
Authors: Berti M; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Ceriotti S; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy., Santi L; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Alberti G; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Beretta S; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Degl'Innocenti S; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Ruatti C; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Savoia EO; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Jofra-Hernandez R; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., De Ponti G; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Bolamperti S; Endocrine and Osteometabolic Laboratory, Institute of Endocrine and Metabolic Sciences, IRCCS San Raffaele Hospital, 20132 Milan, Italy., Villa I; Endocrine and Osteometabolic Laboratory, Institute of Endocrine and Metabolic Sciences, IRCCS San Raffaele Hospital, 20132 Milan, Italy., Galeotti F; UniMoRe: Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy., Romano A; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy., Visigalli I; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Norata R; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Rocchi M; Pathology Unit, IRCCS San Raffaele Hospital, 20132 Milan, Italy., Cristofori P; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Cossutta M; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy., Consiglieri G; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy., Tucci F; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy., Santorelli L; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy., Grumati P; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.; Department of Clinical Medicine and Surgery, Federico II University, 80131 Naples, Italy., Ronfani L; Core Facility for Conditional Mutagenesis, IRCCS San Raffaele Hospital, 20132 Milan, Italy., D'Adamo P; Mouse Behavior Facility, IRCCS San Raffaele Hospital, 20132 Milan, Italy., Giustina A; Endocrine and Osteometabolic Laboratory, Institute of Endocrine and Metabolic Sciences, IRCCS San Raffaele Hospital, 20132 Milan, Italy., Angelozzi M; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy., Settembre C; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.; Department of Clinical Medicine and Surgery, Federico II University, 80131 Naples, Italy., Mortellaro A; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Scala S; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy., Sanvito F; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; Pathology Unit, IRCCS San Raffaele Hospital, 20132 Milan, Italy., Volpi N; UniMoRe: Department of Life Sciences, University of Modena and Reggio Emilia, 41125 Modena, Italy., Aiuti A; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy., Bernardo ME; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy., Crippa S; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.
Source: Disease models & mechanisms [Dis Model Mech] 2026 Feb 01; Vol. 19 (2). Date of Electronic Publication: 2026 Mar 05.
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
Journal Info: Publisher: Company of Biologists Ltd Country of Publication: England NLM ID: 101483332 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1754-8411 (Electronic) Linking ISSN: 17548403 NLM ISO Abbreviation: Dis Model Mech Subsets: MEDLINE
Database: MEDLINE Ultimate
FullText Text:
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PubType: Academic Journal
PubTypeId: academicJournal
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  Data: Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease.
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  Data: <searchLink fieldCode="AU" term="%22Berti+M%22">Berti M</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ceriotti+S%22">Ceriotti S</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Santi+L%22">Santi L</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Alberti+G%22">Alberti G</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Beretta+S%22">Beretta S</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Degl'Innocenti+S%22">Degl'Innocenti S</searchLink>; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Ruatti+C%22">Ruatti C</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Savoia+EO%22">Savoia EO</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Jofra-Hernandez+R%22">Jofra-Hernandez R</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22De+Ponti+G%22">De Ponti G</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Bolamperti+S%22">Bolamperti S</searchLink>; Endocrine and Osteometabolic Laboratory, Institute of Endocrine and Metabolic Sciences, IRCCS San Raffaele Hospital, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Villa+I%22">Villa I</searchLink>; 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Pathology Unit, IRCCS San Raffaele Hospital, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Cristofori+P%22">Cristofori P</searchLink>; Good Laboratory Practice (GLP) Facility, San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Cossutta+M%22">Cossutta M</searchLink>; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Consiglieri+G%22">Consiglieri G</searchLink>; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Tucci+F%22">Tucci F</searchLink>; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Santorelli+L%22">Santorelli L</searchLink>; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.<br /><searchLink fieldCode="AU" term="%22Grumati+P%22">Grumati P</searchLink>; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.; Department of Clinical Medicine and Surgery, Federico II University, 80131 Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Ronfani+L%22">Ronfani L</searchLink>; Core Facility for Conditional Mutagenesis, IRCCS San Raffaele Hospital, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22D'Adamo+P%22">D'Adamo P</searchLink>; Mouse Behavior Facility, IRCCS San Raffaele Hospital, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Giustina+A%22">Giustina A</searchLink>; Endocrine and Osteometabolic Laboratory, Institute of Endocrine and Metabolic Sciences, IRCCS San Raffaele Hospital, 20132 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Angelozzi+M%22">Angelozzi M</searchLink>; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.<br /><searchLink fieldCode="AU" term="%22Settembre+C%22">Settembre C</searchLink>; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.; 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IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Bernardo+ME%22">Bernardo ME</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.; IMedical School, Vita-Salute San Raffaele University, 20132 Milan, Italy.; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Hospital, 41121 Milan, Italy.<br /><searchLink fieldCode="AU" term="%22Crippa+S%22">Crippa S</searchLink>; IRCSS San Raffaele Hospital-San Raffaele Telethon Institute for Gene Therapy (SR-TIGET), 20132 Milan, Italy.
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