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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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ISSN:2296-858X
DOI:10.3389/fmed.2026.1813362