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Antisense oligonucleotide therapy corrects splicing in the common Stargardt disease type 1-causing variant ABCA4 c.5461-10T>C.
Kaltak, Melita; de Bruijn, Petra; Piccolo, Davide; Lee, Sang-Eun; Dulla, Kalyan; Hoogenboezem, Thomas; Beumer, Wouter; Webster, Andrew R; Collin, Rob W J; Cheetham, Michael E; Platenburg, Gerard; Swildens, Jim.
Affiliation
  • Kaltak M; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • de Bruijn P; Department of Human Genetics, Radboud University Medical Center, Geert Grooteplein-Zuid 10, 6525 GA Nijmegen, the Netherlands.
  • Piccolo D; Academic Alliance Genetics, Radboud University Medical Center, Geert Grooteplein-Zuid 10, 6525 GA Nijmegen, and Maastricht University Medical Center+, P. Debyelaan 25, 6229 HX Maastricht, the Netherlands.
  • Lee SE; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Dulla K; UCL, Institute of Ophthalmology, 11-43 Bath Street, EC1V 9EL London, UK.
  • Hoogenboezem T; UCL, Institute of Ophthalmology, 11-43 Bath Street, EC1V 9EL London, UK.
  • Beumer W; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Webster AR; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Collin RWJ; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Cheetham ME; UCL, Institute of Ophthalmology, 11-43 Bath Street, EC1V 9EL London, UK.
  • Platenburg G; Moorfields Eye Hospital, 162 City Road, EC1V 2PD London, UK.
  • Swildens J; Department of Human Genetics, Radboud University Medical Center, Geert Grooteplein-Zuid 10, 6525 GA Nijmegen, the Netherlands.
Mol Ther Nucleic Acids ; 31: 674-688, 2023 Mar 14.
Article in En | MEDLINE | ID: mdl-36910710
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2023 Document type: Article Affiliation country: Netherlands Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Language: En Journal: Mol Ther Nucleic Acids Year: 2023 Document type: Article Affiliation country: Netherlands Country of publication: United States