Journal Article

DKFZ-2024-00891

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Feasibility of Direct Vitrectomy-Sparing Subretinal Injection for Gene Delivery in Large Animals.

Stranak, Z. ; Ardan, T. ; Nemesh, Y. ; Toms, M. ; Toualbi, L. ; Harbottle, R.DKFZ* ; Ellederova, Z. ; Lytvynchuk, L. ; Petrovski, G. ; Motlik, J. ; Moosajee, M. ; Kozak, I.

2024
Taylor & Francis Group Abingdon

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Please use a persistent id in citations: doi:10.1080/02713683.2024.2343335

Abstract: To assess the safety and feasibility of direct vitrectomy-sparing subretinal injection for gene delivery in a large animal model.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.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.This pilot study demonstrates that successful subretinal delivery of gene therapy vectors is achievable using a direct injection without prior vitrectomy surgery.

Keyword(s): Subretinal injection ; large animal ; non-viral gene vector ; pars plana vitrectomy ; safety

Note: 2024 Aug;49(8):879-887

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Contributing Institute(s):

1. DNA-Vektoren (D420)
2. DNA-Vektoren (F160)

Research Program(s):

1. 314 - Immunologie und Krebs (POF4-314) (POF4-314)

Appears in the scientific report 2024

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Database coverage:
; Clarivate Analytics Master Journal List ; Current Contents - Life Sciences ; Ebsco Academic Search ; Essential Science Indicators ; IF < 5 ; JCR ; Nationallizenz ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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