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Related Concept Videos

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

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Related Experiment Video

Updated: Jul 22, 2026

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye
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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.

Current Eye Research
|April 26, 2024
PubMed
Summary

Direct subretinal injection offers a safe and feasible alternative for gene delivery, bypassing the need for vitrectomy surgery. This simplified approach reduces surgical time and recovery, achieving successful gene therapy vector delivery in animal models.

Keywords:
Subretinal injectionlarge animalnon-viral gene vectorpars plana vitrectomysafety

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Area of Science:

  • Ophthalmology
  • Gene Therapy
  • Retinal Surgery

Background:

  • Subretinal gene delivery is crucial for treating retinal diseases.
  • Traditional methods often involve complex procedures like pars plana vitrectomy.

Purpose of the Study:

  • To evaluate the safety and feasibility of direct vitrectomy-sparing subretinal injection for gene delivery.
  • To compare this novel approach with conventional methods in a large animal model.

Main Methods:

  • Liběchov minipigs received subretinal plasmid DNA vector delivery.
  • Eyes were randomized to either direct injection or injection post-vitrectomy (control).
  • Intraoperative and postoperative outcomes were monitored for 30 days.

Main Results:

  • Direct subretinal injection was performed without prior vitrectomy.
  • No adverse events (endophthalmitis, retinal detachment, IOP elevation) were observed.
  • The experimental group showed shorter surgical times and faster recovery.

Conclusions:

  • Direct subretinal injection is a safe and effective method for gene therapy vector delivery.
  • This technique eliminates the need for vitrectomy, simplifying the procedure.
  • This pilot study supports the potential of vitrectomy-sparing injections for retinal gene therapy.