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Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations.
Dulla, Kalyan; Slijkerman, Ralph; van Diepen, Hester C; Albert, Silvia; Dona, Margo; Beumer, Wouter; Turunen, Janne J; Chan, Hee Lam; Schulkens, Iris A; Vorthoren, Lars; den Besten, Cathaline; Buil, Levi; Schmidt, Iris; Miao, Jiayi; Venselaar, Hanka; Zang, Jingjing; Neuhauss, Stephan C F; Peters, Theo; Broekman, Sanne; Pennings, Ronald; Kremer, Hannie; Platenburg, Gerard; Adamson, Peter; de Vrieze, Erik; van Wijk, Erwin.
Affiliation
  • Dulla K; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Slijkerman R; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • van Diepen HC; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Albert S; Department of Human Genetics, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Dona M; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Beumer W; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Turunen JJ; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Chan HL; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Schulkens IA; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Vorthoren L; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • den Besten C; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Buil L; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Schmidt I; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Miao J; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Venselaar H; Center for Molecular and Biomolecular Informatics, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Zang J; University of Zürich, Institute of Molecular Life Sciences, 8057 Zürich, Switzerland.
  • Neuhauss SCF; University of Zürich, Institute of Molecular Life Sciences, 8057 Zürich, Switzerland.
  • Peters T; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Broekman S; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Pennings R; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • Kremer H; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands; Department of Human Genetics, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the
  • Platenburg G; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Adamson P; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands; UCL, Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
  • de Vrieze E; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands.
  • van Wijk E; Department of Otorhinolaryngology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 GA Nijmegen, the Netherlands. Electronic address: erwin.vanwyk@radboudumc.nl.
Mol Ther ; 29(8): 2441-2455, 2021 08 04.
Article in En | MEDLINE | ID: mdl-33895329
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Retinitis Pigmentosa / Extracellular Matrix Proteins / Oligonucleotides, Antisense / Mutation Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2021 Type: Article Affiliation country: Netherlands
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Retinitis Pigmentosa / Extracellular Matrix Proteins / Oligonucleotides, Antisense / Mutation Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2021 Type: Article Affiliation country: Netherlands