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Updated: May 30, 2026

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models

Published on: March 16, 2022

Polymeric vectors for ocular gene delivery.

Viral Tamboli¹, Gyan P Mishra, Ashim K Mitrat

¹Division of Pharmaceutical Sciences, School of Pharmacy, University of Missouri-Kansas City, 2464 Charlotte Street, Kansas City, MO 64108-2718, USA.

Therapeutic Delivery

|August 23, 2011

Summary

This summary is machine-generated.

Polymeric vectors offer a promising, non-toxic approach for ocular gene therapy. These nanoparticles facilitate gene delivery by overcoming cellular barriers, enhancing treatment for eye diseases.

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

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Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry

Published on: March 1, 2013

Area of Science:

Ophthalmology
Biotechnology
Gene Therapy

Background:

Gene therapy shows potential for treating inherited and acquired eye diseases.
Efficient gene transfer to target cells with minimal toxicity is crucial for ocular gene therapy success.
Intracellular and extracellular barriers pose significant challenges for ocular gene delivery.

Purpose of the Study:

To review synthetic and natural polymeric systems for ocular gene delivery.
To highlight the advantages of polymeric vectors in overcoming gene delivery barriers.
To discuss the potential of chemically modified polymers for enhanced gene delivery.

Main Methods:

Exploration of viral and nonviral vectors for improved transfection efficiency.
Focus on nonviral delivery systems, particularly polymeric vectors.
Preparation of polyplexes (nanoparticles) from cationic polymers and DNA.

Main Results:

Polymeric vectors are nontoxic and non-immunogenic, making them attractive for ocular gene delivery.
Polyplexes facilitate cellular uptake, endolysosomal escape, and nuclear entry.
Chemical modification of polymers allows for tailored delivery vector properties.

Conclusions:

Polymeric vectors represent a significant advancement in nonviral ocular gene delivery.
These systems effectively address barriers to gene transfer in the eye.
Further development of synthetic and natural polymeric systems holds promise for future eye disease treatments.