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RNA activation as a precision dosing modality: MTL-CEBPA for controlled enzyme elevation in MPS I-H.

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Title: RNA activation as a precision dosing modality: MTL-CEBPA for controlled enzyme elevation in MPS I-H.
Authors: Reebye V; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.; Dawn Therapeutics Limited, London, United Kingdom., Skourti-Stathaki K; MiNA Therapeutics Limited, London, United Kingdom., Vanezis K; MiNA Therapeutics Limited, London, United Kingdom., Song M; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States., Przybilla MJ; Division of Pediatric Blood and Bone Marrow Transplantation, Minneapolis, MN, United States., Purdie K; MiNA Therapeutics Limited, London, United Kingdom., Tesone AJ; MiNA Therapeutics Limited, London, United Kingdom., Pizza G; MiNA Therapeutics Limited, London, United Kingdom., Jarvis S; Department of Surgery and Cancer, Imperial College London, London, United Kingdom., Raulf N; MiNA Therapeutics Limited, London, United Kingdom., Andrikakou P; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom., Vasara J; MiNA Therapeutics Limited, London, United Kingdom., Setten R; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States., Nicholls J; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.; Dawn Therapeutics Limited, London, United Kingdom., Hegre SA; Department of Cancer Research and Molecular Medicine, Norwegian University of Science and Technology, Trondheim, Norway.; Department of Computer and Information Science, Norwegian University of Science and Technology, Trondheim, Norway., Sætrom P; Department of Cancer Research and Molecular Medicine, Norwegian University of Science and Technology, Trondheim, Norway.; Department of Computer and Information Science, Norwegian University of Science and Technology, Trondheim, Norway., Habib R; MiNA Therapeutics Limited, London, United Kingdom., Huang KW; National Taiwan University Hospital, Taipei, Taiwan., Farzaneh F; The Rayne Institute, King's College London, London, United Kingdom., Li H; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States., Rossi JJ; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States., Tomatsu S; Skeletal Dysplasia Research Laboratory, Nemours/Alfred I DuPont Hospital for Children, Wilmington, DE, United States., Khan S; Skeletal Dysplasia Research Laboratory, Nemours/Alfred I DuPont Hospital for Children, Wilmington, DE, United States., Furness L; Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University NHS Foundation Trust, Manchester, United Kingdom., Wynn R; Department of Pediatric Bone Marrow Transplantation, Manchester University NHS Foundation Trust, Manchester, United Kingdom., Whitley CB; Division of Pediatric Blood and Bone Marrow Transplantation, Minneapolis, MN, United States., Jones SA; Department of Pediatric Bone Marrow Transplantation, Manchester University NHS Foundation Trust, Manchester, United Kingdom., Habib NA; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.; Dawn Therapeutics Limited, London, United Kingdom.
Source: Frontiers in medicine [Front Med (Lausanne)] 2026 May 07; Vol. 13, pp. 1813362. Date of Electronic Publication: 2026 May 07 (Print Publication: 2026).
Publication Type: Journal Article
Journal Info: Publisher: Frontiers Media S.A Country of Publication: Switzerland NLM ID: 101648047 Publication Model: eCollection Cited Medium: Print ISSN: 2296-858X (Print) Linking ISSN: 2296858X NLM ISO Abbreviation: Front Med (Lausanne) Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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  Data: RNA activation as a precision dosing modality: MTL-CEBPA for controlled enzyme elevation in MPS I-H.
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  Data: <searchLink fieldCode="AU" term="%22Reebye+V%22">Reebye V</searchLink>; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.; Dawn Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Skourti-Stathaki+K%22">Skourti-Stathaki K</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Vanezis+K%22">Vanezis K</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Song+M%22">Song M</searchLink>; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States.<br /><searchLink fieldCode="AU" term="%22Przybilla+MJ%22">Przybilla MJ</searchLink>; Division of Pediatric Blood and Bone Marrow Transplantation, Minneapolis, MN, United States.<br /><searchLink fieldCode="AU" term="%22Purdie+K%22">Purdie K</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Tesone+AJ%22">Tesone AJ</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Pizza+G%22">Pizza G</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Jarvis+S%22">Jarvis S</searchLink>; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Raulf+N%22">Raulf N</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Andrikakou+P%22">Andrikakou P</searchLink>; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Vasara+J%22">Vasara J</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Setten+R%22">Setten R</searchLink>; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States.<br /><searchLink fieldCode="AU" term="%22Nicholls+J%22">Nicholls J</searchLink>; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.; Dawn Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Hegre+SA%22">Hegre SA</searchLink>; Department of Cancer Research and Molecular Medicine, Norwegian University of Science and Technology, Trondheim, Norway.; Department of Computer and Information Science, Norwegian University of Science and Technology, Trondheim, Norway.<br /><searchLink fieldCode="AU" term="%22Sætrom+P%22">Sætrom P</searchLink>; Department of Cancer Research and Molecular Medicine, Norwegian University of Science and Technology, Trondheim, Norway.; Department of Computer and Information Science, Norwegian University of Science and Technology, Trondheim, Norway.<br /><searchLink fieldCode="AU" term="%22Habib+R%22">Habib R</searchLink>; MiNA Therapeutics Limited, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Huang+KW%22">Huang KW</searchLink>; National Taiwan University Hospital, Taipei, Taiwan.<br /><searchLink fieldCode="AU" term="%22Farzaneh+F%22">Farzaneh F</searchLink>; The Rayne Institute, King's College London, London, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Li+H%22">Li H</searchLink>; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States.<br /><searchLink fieldCode="AU" term="%22Rossi+JJ%22">Rossi JJ</searchLink>; Department of Molecular & Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA, United States.<br /><searchLink fieldCode="AU" term="%22Tomatsu+S%22">Tomatsu S</searchLink>; Skeletal Dysplasia Research Laboratory, Nemours/Alfred I DuPont Hospital for Children, Wilmington, DE, United States.<br /><searchLink fieldCode="AU" term="%22Khan+S%22">Khan S</searchLink>; Skeletal Dysplasia Research Laboratory, Nemours/Alfred I DuPont Hospital for Children, Wilmington, DE, United States.<br /><searchLink fieldCode="AU" term="%22Furness+L%22">Furness L</searchLink>; Manchester Centre for Genomic Medicine, St Mary's Hospital, Manchester University NHS Foundation Trust, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Wynn+R%22">Wynn R</searchLink>; Department of Pediatric Bone Marrow Transplantation, Manchester University NHS Foundation Trust, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Whitley+CB%22">Whitley CB</searchLink>; Division of Pediatric Blood and Bone Marrow Transplantation, Minneapolis, MN, United States.<br /><searchLink fieldCode="AU" term="%22Jones+SA%22">Jones SA</searchLink>; Department of Pediatric Bone Marrow Transplantation, Manchester University NHS Foundation Trust, Manchester, United Kingdom.<br /><searchLink fieldCode="AU" term="%22Habib+NA%22">Habib NA</searchLink>; Department of Surgery and Cancer, Imperial College London, London, United Kingdom.; MiNA Therapeutics Limited, London, United Kingdom.; Dawn Therapeutics Limited, London, United Kingdom.
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