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. |
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| 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 |
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