Rescue of the MERTK phagocytic defect in a human iPSC disease model using translational read-through inducing drugs.

Autor: Ramsden CM; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, EC1V 2PD, UK.; Moorfields Eye Hospital NHS Foundation Trust, 162 City Road, London, EC1V 2PD, UK., Nommiste B; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK., R Lane A; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK., Carr AF; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK., Powner MB; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK.; Division of Optometry and Visual Sciences, City University London, Northampton Square, London, EC1V 0HB, UK., J K Smart M; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK., Chen LL; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK., Muthiah MN; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, EC1V 2PD, UK.; Moorfields Eye Hospital NHS Foundation Trust, 162 City Road, London, EC1V 2PD, UK., Webster AR; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, EC1V 2PD, UK., Moore AT; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, EC1V 2PD, UK.; UCSF School of Medicine, Koret Vision Center, 10 Koret Way, San Francisco, CA 94143, USA., Cheetham ME; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK., da Cruz L; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, EC1V 2PD, UK.; Moorfields Eye Hospital NHS Foundation Trust, 162 City Road, London, EC1V 2PD, UK., Coffey PJ; Department of Ocular Biology and Therapeutics (ORBIT), Institute of Ophthalmology, University College London, 11-43 Bath Street, London, EC1V 9EL, UK. p.coffey@ucl.ac.uk.; NIHR Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology, London, EC1V 2PD, UK. p.coffey@ucl.ac.uk.; Center for Stem Cell Biology and Engineering, NRI, UC Santa Barbara, USA. p.coffey@ucl.ac.uk.

Publikováno v: Scientific reports [Sci Rep] 2017 Mar 03; Vol. 7 (1), pp. 51. Date of Electronic Publication: 2017 Mar 03.