Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington disease models.

Autor: Sogorb-Gonzalez M; Department of Research & Development, uniQure Biopharma B.V., 1105BP, Amsterdam, The Netherlands.; Department of Gastroenterology and Hepatology, Leiden University Medical Center, 2333 ZA, Leiden, The Netherlands., Landles C; Huntington's Disease Centre, Department of Neurodegenerative Disease and UK Dementia Research Institute at UCL, Queen Square Institute of Neurology, UCL, Queen Square, WC1N 3BG, London, UK., Caron NS; Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, University of British Columbia, V5Z 4H4, Vancouver, Canada., Stam A; Department of Research & Development, uniQure Biopharma B.V., 1105BP, Amsterdam, The Netherlands., Osborne G; Huntington's Disease Centre, Department of Neurodegenerative Disease and UK Dementia Research Institute at UCL, Queen Square Institute of Neurology, UCL, Queen Square, WC1N 3BG, London, UK., Hayden MR; Centre for Molecular Medicine and Therapeutics, Department of Medical Genetics, University of British Columbia, V5Z 4H4, Vancouver, Canada., Howland D; CHDI Management/CHDI Foundation, NJ 08540, Princeton, USA., van Deventer S; Department of Gastroenterology and Hepatology, Leiden University Medical Center, 2333 ZA, Leiden, The Netherlands., Bates GP; Huntington's Disease Centre, Department of Neurodegenerative Disease and UK Dementia Research Institute at UCL, Queen Square Institute of Neurology, UCL, Queen Square, WC1N 3BG, London, UK., Vallès A; Department of Research & Development, uniQure Biopharma B.V., 1105BP, Amsterdam, The Netherlands., Evers M; Department of Research & Development, uniQure Biopharma B.V., 1105BP, Amsterdam, The Netherlands.

Publikováno v: Brain : a journal of neurology [Brain] 2024 Aug 18. Date of Electronic Publication: 2024 Aug 18.

Early clinical outcomes of gunshot isolated fibular fractures treated non-operatively at an urban academic hospital

Autor: Nkosi, C.S., Maseko, M., Sekeitto, A.R., van Deventer, S.

Publikováno v: East African Orthopaedic Journal; Vol. 17 No. 1 (2023); 3-10

Objectives: To report the outcomes of wound infection and to determine the outcomes of fracture union. Methods: A retrospective review of patients who sustained gunshot isolated fibular shaft fractures that were treated non-operatively with antibioti

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=78975075580c::6e240b01f50a7121f5fa0522abcf79c6
https://www.ajol.info/index.php/eaoj/article/view/249394

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Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery

Autor: Pietersz, K. L., Martier, R. M., Baatje, M. S., Liefhebber, J. M., Brouwers, C. C., Pouw, S. M., Fokkert, L., Lubelski, J., Petry, H., Martens, G. J. M., van Deventer, S. J., Konstantinova, P., Blits, B.

Publikováno v: Gene Therapy; August 2021, Vol. 28 Issue: 7-8 p435-446, 12p

Mapping the European startup landscape.

Autor: van Wilgenburg B; Forbion Capital Partners, Naarden, the Netherlands., van Wilgenburg K; Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands., Paisner K; KP2 LLC, Oakland, CA, USA., van Deventer S; Forbion Capital Partners, Naarden, the Netherlands., Rooswinkel RW; Forbion Capital Partners, Naarden, the Netherlands. rogier.rooswinkel@forbion.com.

Publikováno v: Nature biotechnology [Nat Biotechnol] 2019 Apr; Vol. 37 (4), pp. 345-349.