Reference
Michaelides, M., Laich, Y., Wong, S. C., Oluonye, N., Zaman, S., Kumaran, N., Kalitzeos, A., Petrushkin, H., Georgiou, M., Tailor, V., Pabst, M., Staeubli, K., Maimon-Mor, R. O., Jones, P. R., Scholte, S. H., Georgiadis, A., Spuy, J. van der, Naylor, S., Forbes, A., … Bainbridge, J. W. B. (2025). Gene therapy in children with AIPL1-associated severe retinal dystrophy: An open-label, first-in-human interventional study. The Lancet, 405(10479), 648–657. https://doi.org/10.1016/S0140-6736(24)02812-5
Info
FirstAuthor:: Michaelides, Michel
Author:: Laich, Yannik
Author:: Wong, Sui Chien
Author:: Oluonye, Ngozi
Author:: Zaman, Serena
Author:: Kumaran, Neruban
Author:: Kalitzeos, Angelos
Author:: Petrushkin, Harry
Author:: Georgiou, Michalis
Author:: Tailor, Vijay
Author:: Pabst, Marc
Author:: Staeubli, Kim
Author:: Maimon-Mor, Roni O.
Author:: Jones, Peter R.
Author:: Scholte, Steven H.
Author:: Georgiadis, Anastasios
Author:: Spuy, Jacqueline van der
Author:: Naylor, Stuart
Author:: Forbes, Alexandria
Author:: Dekker, Tessa M.
Author:: Arulmuthu, Eugene R.
Author:: Smith, Alexander J.
Author:: Ali, Robin R.
Author:: Bainbridge, James W. B.
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Title:: Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
Year:: 2025
Citekey:: MichaelidesEtAl_2025_GeneTherapyChildren
itemType:: journalArticle
Journal:: The Lancet
Volume:: 405
Issue:: 10479
Pages:: 648-657
DOI:: 10.1016/S0140-6736(24)02812-5
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Abstract
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This non-randomised, single-arm, clinical study conducted in the UK involved four children aged 1·0–2·8 years with severe retinal dystrophy associated with biallelic disease-causing sequence variants in AIPL1.