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Three-Year Safety Results of SAR422459 (EIAV-ABCA4) Gene Therapy in Patients With ABCA4-Associated Stargardt Disease: An Open-Label Dose-Escalation Phase I/IIa Clinical Trial, Cohorts 1-5.
Parker MA, Erker LR, Audo I, Choi D, Mohand-Said S, Sestakauskas K, Benoit P, Appelqvist T, Krahmer M, Ségaut-Prévost C, Lujan BJ, Faridi A, Chegarnov EN, Steinkamp PN, Ku C, da Palma MM, Barale PO, Ayelo-Scheer S, Lauer A, Stout T, Wilson DJ, Weleber RG, Pennesi ME, Sahel JA, Yang P. Parker MA, et al. Among authors: ku c. Am J Ophthalmol. 2022 Aug;240:285-301. doi: 10.1016/j.ajo.2022.02.013. Epub 2022 Mar 4. Am J Ophthalmol. 2022. PMID: 35248547 Free PMC article. Clinical Trial.
Molecular and Clinical Findings in Patients With Knobloch Syndrome.
Hull S, Arno G, Ku CA, Ge Z, Waseem N, Chandra A, Webster AR, Robson AG, Michaelides M, Weleber RG, Davagnanam I, Chen R, Holder GE, Pennesi ME, Moore AT. Hull S, et al. JAMA Ophthalmol. 2016 Jul 1;134(7):753-62. doi: 10.1001/jamaophthalmol.2016.1073. JAMA Ophthalmol. 2016. PMID: 27259167
Detailed Clinical Phenotype and Molecular Genetic Findings in CLN3-Associated Isolated Retinal Degeneration.
Ku CA, Hull S, Arno G, Vincent A, Carss K, Kayton R, Weeks D, Anderson GW, Geraets R, Parker C, Pearce DA, Michaelides M, MacLaren RE, Robson AG, Holder GE, Heon E, Raymond FL, Moore AT, Webster AR, Pennesi ME. Ku CA, et al. JAMA Ophthalmol. 2017 Jul 1;135(7):749-760. doi: 10.1001/jamaophthalmol.2017.1401. JAMA Ophthalmol. 2017. PMID: 28542676 Free PMC article.
The new landscape of retinal gene therapy.
Ku CA, Pennesi ME. Ku CA, et al. Am J Med Genet C Semin Med Genet. 2020 Sep;184(3):846-859. doi: 10.1002/ajmg.c.31842. Epub 2020 Sep 5. Am J Med Genet C Semin Med Genet. 2020. PMID: 32888388 Review.
843 results