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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Related Experiment Video

Updated: Feb 3, 2026

Optimized Minimally Invasive Transscleral Subretinal Injection Technique in Mouse
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Optimized Subretinal Injection Technique for Gene Therapy Approaches.

Regine Mühlfriedel1, Stylianos Michalakis2, Marina Garcia Garrido3

  • 1Division of Ocular Neurodegeneration, Institute for Ophthalmic Research, Centre for Ophthalmology, Eberhard Karls Universität Tübingen, Tübingen, Germany. regine.muehlfriedel@med.uni-tuebingen.de.

Methods in Molecular Biology (Clifton, N.J.)
|October 17, 2018
PubMed
Summary

Transscleral pars plana virus delivery offers a refined method for gene therapy in small mouse eyes, minimizing procedure-related complications for inherited eye diseases.

Keywords:
AblationBleb formationGene deliveryIn vivo imaging techniques (SLO SD-OCT)Mouse eyePhotoreceptor cellRecombinant adeno-associated virusRetinaSubretinal injectionTransscleral pars plana injection

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Area of Science:

  • Ophthalmology
  • Gene Therapy
  • Preclinical Research

Background:

  • Inherited eye diseases necessitate local viral vector delivery via intraocular injection.
  • Small animal models, particularly mice, are crucial for preclinical gene therapy studies due to the lack of large animal models.
  • Subretinal injection in mouse eyes can cause adverse effects that confound therapeutic outcomes.

Purpose of the Study:

  • To detail a transscleral pars plana virus-mediated gene transfer method for mouse eyes.
  • To optimize gene transfer in small animal models for inherited eye diseases.
  • To mitigate adverse effects associated with traditional subretinal delivery.

Main Methods:

  • Development of a transscleral pars plana injection technique.
  • Application of viral vectors for gene transfer in mouse eyes.
  • Evaluation of the delivery method's efficacy and safety in small eyes.

Main Results:

  • Successful demonstration of transscleral pars plana gene transfer in mouse eyes.
  • Reduced procedural complications compared to subretinal injections.
  • Potential for improved therapeutic outcomes in preclinical models.

Conclusions:

  • Transscleral pars plana delivery is a viable alternative for gene therapy in mouse models of inherited eye diseases.
  • This method enhances the reliability of preclinical studies by minimizing surgical trauma.
  • Optimized gene transfer in small eyes is achievable through this refined technique.