A phase 1 clinical trial shows safe, sustained, AAV-mediated expression of IL-1Ra in the human osteoarthritic knee joint.

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Title: A phase 1 clinical trial shows safe, sustained, AAV-mediated expression of IL-1Ra in the human osteoarthritic knee joint.
Authors: De la Vega RE; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Department of Orthopedic Surgery, Mayo Clinic, Rochester, MN 55905, USA.; Musculoskeletal Gene Therapy Laboratory, Mayo Clinic, Rochester, MN 55905, USA., Sellon JL; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Department of Orthopedic Surgery, Mayo Clinic, Rochester, MN 55905, USA., Smith J; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA., Wisniewski SJ; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA., Jurisson ML; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA., Frick MA; Department of Radiology, Mayo Clinic, Rochester, MN 55905, USA., Scrabeck TL; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA., Block JB; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA., Mills CJ; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Department of Orthopedic Surgery, Mayo Clinic, Rochester, MN 55905, USA., Pohlkamp ZW; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA., Coenen MJ; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Musculoskeletal Gene Therapy Laboratory, Mayo Clinic, Rochester, MN 55905, USA., Hawse GP; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Musculoskeletal Gene Therapy Laboratory, Mayo Clinic, Rochester, MN 55905, USA., Abdul TY; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Musculoskeletal Gene Therapy Laboratory, Mayo Clinic, Rochester, MN 55905, USA.; Department of Clinical Pharmacology, Mayo Clinic, Rochester, MN 55905, USA., Conaghan P; Leeds Institute of Rheumatic and Musculoskeletal Medicine, University of Leeds and NIHR Leeds Biomedical Research Centre, Leeds LS7 4SA, UK., Keravala A; Genascence Corp., Palo Alto, CA 94306, USA., Chalberg TW; Genascence Corp., Palo Alto, CA 94306, USA., Robbins PD; Institute on the Biology of Aging and Metabolism, Department of Biochemistry, Molecular Biology and Biophysics, University of Minnesota, Minneapolis, MN 55455, USA., Ghivizzani SC; Department of Orthopedics and Rehabilitation, University of Florida College of Medicine, Gainesville, FL 32610, USA., Evans CH; Department of Physical Medicine and Rehabilitation, Mayo Clinic, Rochester, MN 55905, USA.; Department of Orthopedic Surgery, Mayo Clinic, Rochester, MN 55905, USA.; Musculoskeletal Gene Therapy Laboratory, Mayo Clinic, Rochester, MN 55905, USA.; Department of Molecular Medicine, Mayo Clinic, Rochester, MN 55905, USA.
Source: Science translational medicine [Sci Transl Med] 2025 Jun 04; Vol. 17 (801), pp. eadu9804. Date of Electronic Publication: 2025 Jun 04.
Publication Type: Journal Article; Clinical Trial, Phase I
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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ISSN:1946-6242
DOI:10.1126/scitranslmed.adu9804