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Antisense Therapy for a Common Corneal Dystrophy Ameliorates TCF4 Repeat Expansion-Mediated Toxicity.
Zarouchlioti, Christina; Sanchez-Pintado, Beatriz; Hafford Tear, Nathaniel J; Klein, Pontus; Liskova, Petra; Dulla, Kalyan; Semo, Ma'ayan; Vugler, Anthony A; Muthusamy, Kirithika; Dudakova, Lubica; Levis, Hannah J; Skalicka, Pavlina; Hysi, Pirro; Cheetham, Michael E; Tuft, Stephen J; Adamson, Peter; Hardcastle, Alison J; Davidson, Alice E.
Affiliation
  • Zarouchlioti C; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Sanchez-Pintado B; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Hafford Tear NJ; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Klein P; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Liskova P; Research Unit for Rare Diseases, Department of Paediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, Prague 128 08, Czech Republic; Department of Ophthalmology, First Faculty of Medicine, Charles University and Gen
  • Dulla K; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Semo M; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Vugler AA; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Muthusamy K; UCL Institute of Ophthalmology, London ECIV 9EL, UK; Moorfields Eye Hospital, London EC1V 2PD, UK.
  • Dudakova L; Research Unit for Rare Diseases, Department of Paediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, Prague 128 08, Czech Republic.
  • Levis HJ; Institute of Aging and Chronic Disease, University of Liverpool, Liverpool L7 8TX, UK.
  • Skalicka P; Research Unit for Rare Diseases, Department of Paediatrics and Adolescent Medicine, First Faculty of Medicine, Charles University and General University Hospital in Prague, Ke Karlovu 2, Prague 128 08, Czech Republic; Department of Ophthalmology, First Faculty of Medicine, Charles University and Gen
  • Hysi P; Department of Ophthalmology and Twin Research, King's College London, London SE1 7EH, UK.
  • Cheetham ME; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Tuft SJ; UCL Institute of Ophthalmology, London ECIV 9EL, UK; Moorfields Eye Hospital, London EC1V 2PD, UK.
  • Adamson P; ProQR Therapeutics, Zernikedreef 9, 2333 CK Leiden, the Netherlands.
  • Hardcastle AJ; UCL Institute of Ophthalmology, London ECIV 9EL, UK.
  • Davidson AE; UCL Institute of Ophthalmology, London ECIV 9EL, UK. Electronic address: alice.davidson@ucl.ac.uk.
Am J Hum Genet ; 102(4): 528-539, 2018 04 05.
Article in En | MEDLINE | ID: mdl-29526280
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Fuchs' Endothelial Dystrophy / Oligonucleotides, Antisense / Trinucleotide Repeat Expansion / Genetic Predisposition to Disease / Transcription Factor 4 Type of study: Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Aged / Animals / Female / Humans / Male Language: En Journal: Am J Hum Genet Year: 2018 Document type: Article Affiliation country: Reino Unido Country of publication: Estados Unidos
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Fuchs' Endothelial Dystrophy / Oligonucleotides, Antisense / Trinucleotide Repeat Expansion / Genetic Predisposition to Disease / Transcription Factor 4 Type of study: Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limits: Aged / Animals / Female / Humans / Male Language: En Journal: Am J Hum Genet Year: 2018 Document type: Article Affiliation country: Reino Unido Country of publication: Estados Unidos