Stranak, Zbynek;
Ardan, Taras;
Nemesh, Yaroslav;
Toms, Maria;
Toualbi, Lyes;
Harbottle, Richard;
Ellederova, Zdenka;
... Kozak, Igor; + view all
(2024)
Feasibility of Direct Vitrectomy-Sparing Subretinal Injection for Gene Delivery in Large Animals.
Current Eye Research
, 49
(8)
pp. 879-887.
10.1080/02713683.2024.2343335.
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Abstract
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.
| Type: | Article |
|---|---|
| Title: | Feasibility of Direct Vitrectomy-Sparing Subretinal Injection for Gene Delivery in Large Animals |
| Location: | England |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1080/02713683.2024.2343335 |
| Publisher version: | https://doi.org/10.1080/02713683.2024.2343335 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Subretinal injection; pars planavitrectomy; large animal; non-viralgene vector; safety |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > Institute of Ophthalmology |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10203088 |
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