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Gene Therapy

Gene Therapy

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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Updated: Apr 18, 2026

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

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

Published on: August 7, 2015

Nanoparticle-based technologies for retinal gene therapy.

Jeffrey Adijanto¹, Muna I Naash¹

¹University of Oklahoma Health Sciences Center, Department of Cell Biology, Oklahoma City, OK, USA.

European Journal of Pharmaceutics and Biopharmaceutics : Official Journal of Arbeitsgemeinschaft Fur Pharmazeutische Verfahrenstechnik E.V

|January 17, 2015

Summary

This summary is machine-generated.

Non-viral nanoparticles (NPs) offer promising alternatives to adeno-associated virus (AAV) gene therapy for hereditary retinal diseases. This review explores NP biochemical features, cellular uptake, and strategies for sustained gene expression in retinal gene therapy.

Keywords:

CK30 Nanoparticles Non-viral retinal gene therapy PLGA Polylysine Retinal disease Vector engineering

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

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Published on: August 7, 2015

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

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

Published on: November 25, 2012

Area of Science:

Ophthalmology
Gene Therapy
Nanotechnology

Background:

Hereditary retinal diseases cause degeneration, necessitating novel therapeutic strategies.
Adeno-associated virus (AAV) vectors are currently dominant in retinal gene therapy.
Non-viral nanoparticles (NPs) are emerging as alternative gene delivery systems.

Purpose of the Study:

To review the biochemical properties of various non-viral nanoparticles for retinal gene delivery.
To discuss NP cellular internalization, endosomal escape, and nuclear delivery mechanisms.
To highlight successful in vivo NP gene delivery to the retina and retinal pigment epithelium (RPE).

Main Methods:

Review of existing literature on non-viral nanoparticle technologies.
Analysis of NP biochemical features influencing cellular uptake and intracellular trafficking.
Examination of strategies for optimizing gene expression from plasmid DNA.

Main Results:

Different NP categories (polymer, liposomes, peptide compacted DNA) exhibit distinct characteristics.
Key NP features affect cellular internalization, endosomal escape, and nuclear translocation.
Successful in vivo retinal gene delivery using various NPs has been demonstrated in preclinical models.

Conclusions:

Non-viral nanoparticles present viable therapeutic options for retinal gene therapy.
Understanding NP-cell interactions is crucial for optimizing delivery efficiency.
Strategies exist to achieve persistent, high-level gene expression for treating retinal degeneration.