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Updated: Jul 22, 2026
Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
Published on: November 25, 2012
Feasibility of Direct Vitrectomy-Sparing Subretinal Injection for Gene Delivery in Large Animals
Zbynek Stranak1, Taras Ardan2, Yaroslav Nemesh2
1Department of Ophthalmology, Charles University, Prague and the Kralovske Vinohrady University Hospital, Prague, Czech Republic.
Purpose:
To assess the safety and feasibility of direct vitrectomy-sparing subretinal injection for gene delivery in a large animal model.
Methods:
The experimental Liběchov minipigs were used for subretinal delivery of a plasmid DNA vector (pS/MAR-CMV-copGFP) with cytomegalovirus (CMV) promoter, green fluorescent protein (GFP) reporter (copGFP) and a scaffold/matrix attachment region (S/MAR) sequence. The eyes were randomized to subretinal injection of the vector following pars plana vitrectomy (control group) or a direct injection without prior vitrectomy surgery (experimental group). Intra- and post-operative observations up to 30 days after surgery were compared.
Results:
Six eyes of three mini-pigs underwent surgery for delivery into the subretinal space. Two eyes in the control group were operated with a classical approach (lens-sparing vitrectomy and posterior hyaloid detachment). The other four eyes in the experimental group were injected directly with a subretinal cannula without vitrectomy surgery. No adverse events, such as endophthalmitis, retinal detachment and intraocular pressure elevation were observed post-operatively. The eyes in the experimental group had both shorter surgical time and recovery while achieving the same surgical goal.
Conclusions:
This pilot study demonstrates that successful subretinal delivery of gene therapy vectors is achievable using a direct injection without prior vitrectomy surgery.
