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Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice.
Marangoni D, Bush RA, Zeng Y, Wei LL, Ziccardi L, Vijayasarathy C, Bartoe JT, Palyada K, Santos M, Hiriyanna S, Wu Z, Colosi P, Sieving PA. Marangoni D, et al. Among authors: hiriyanna s. Mol Ther Methods Clin Dev. 2016 Mar 16;5:16011. doi: 10.1038/mtm.2016.11. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27626041 Free PMC article.
Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.
Cukras C, Wiley HE, Jeffrey BG, Sen HN, Turriff A, Zeng Y, Vijayasarathy C, Marangoni D, Ziccardi L, Kjellstrom S, Park TK, Hiriyanna S, Wright JF, Colosi P, Wu Z, Bush RA, Wei LL, Sieving PA. Cukras C, et al. Among authors: hiriyanna s. Mol Ther. 2018 Sep 5;26(9):2282-2294. doi: 10.1016/j.ymthe.2018.05.025. Epub 2018 Jul 7. Mol Ther. 2018. PMID: 30196853 Free PMC article. Clinical Trial.
32 results