Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice

Dario Marangoni, Ronald A. Bush, Yong Zeng, Lisa L. Wei, Lucia Ziccardi, Camasamudram Vijayasarathy, Joshua T. Bartoe, Kiran Palyada, Maria Santos, Suja Hiriyanna, Zhijian Wu, Peter Colosi, Paul A. Sieving

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15 Scopus citations

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Medicine & Life Sciences