Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

Author:

Cukras Catherine,Wiley Henry E.,Jeffrey Brett G.,Sen H. Nida,Turriff Amy,Zeng Yong,Vijayasarathy Camasamudram,Marangoni Dario,Ziccardi Lucia,Kjellstrom Sten,Park Tae Kwon,Hiriyanna Suja,Wright J. Fraser,Colosi Peter,Wu Zhijian,Bush Ronald A.,Wei Lisa L.,Sieving Paul A.

Funder

National Institutes of Health

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference47 articles.

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3. Evaluation of patients with obvious or suspected degenerative and dystrophic disorders of the retina, pigment epithelium and choroid. Experience from a Swedish referral center;Skoog;Acta Ophthalmol. (Copenh.),1990

4. X linked retinoschisis;George;Br. J. Ophthalmol.,1995

5. Synaptic pathology and therapeutic repair in adult retinoschisis mouse by AAV-RS1 transfer;Ou;J. Clin. Invest.,2015

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