Advertisement

Topics

Lipid nanoparticles for delivery of messenger RNA to the back of the eye.

08:00 EDT 12th April 2019 | BioPortfolio

Summary of "Lipid nanoparticles for delivery of messenger RNA to the back of the eye."

Retinal gene therapy has had unprecedented success in generating treatments that can halt vision loss. However, immunogenic response and long-term toxicity with the use of viral vectors remain a concern. Non-viral vectors are relatively non-immunogenic, scalable platforms that have limited success with DNA delivery to the eye. Messenger RNA (mRNA) therapeutics has expanded the ability to achieve high gene expression while eliminating unintended genomic integration or the need to cross the restrictive nuclear barrier. Lipid-based nanoparticles (LNPs) remain at the forefront of potent delivery vectors for nucleic acids. Herein, we tested eleven different LNP variants for their ability to deliver mRNA to the back of the eye. LNPs that contained ionizable lipids with low pKa and unsaturated hydrocarbon chains showed the highest amount of a reporter gene transfection in the retina. The kinetics of gene expression showed a rapid onset (within 4 h) that persisted for 96 h. The gene delivery was cell-type specific with majority of the expression in the retinal pigmented epithelium (RPE) and limited expression in the Müller glia. LNP-delivered mRNA can be used to treat monogenic retinal degenerative disorders of the RPE. The transient nature of mRNA-based therapeutics makes it desirable for applications that are directed towards retinal reprogramming or genome editing. Overall, non-viral delivery of RNA therapeutics to diverse cell types within the retina can provide transformative new approaches to prevent blindness.

Affiliation

Journal Details

This article was published in the following journal.

Name: Journal of controlled release : official journal of the Controlled Release Society
ISSN: 1873-4995
Pages:

Links

DeepDyve research library

PubMed Articles [10649 Associated PubMed Articles listed on BioPortfolio]

Potential of Lipid Nanoparticles (SLNs and NLCs) in Enhancing Oral Bioavailability of Drugs with Poor Intestinal Permeability.

Lipid-based drug delivery systems has become a popular choice for oral delivery of lipophilic drugs with dissolution rate limited oral absorption. Lipids are known to enhance oral bioavailability of p...

Solid lipid nanoparticles for ocular delivery of isoniazid: evaluation, proof of concept and in vivo safety & kinetics.

Evaluation of solid lipid nanoparticles (SLNs) for ocular delivery of isoniazid (INH).

Nano-scale drug delivery systems for antiarrhythmic agents.

Arrhythmia means the heart is beating too fast, too slow, or with an irregular pattern. Due to the side effects and low bioavailability of many antiarrhythmic drugs, nano-encapsulation has been widely...

Solid Lipid Nanoparticles for Targeted Delivery of Triclosan into Skin for Infection Prevention.

Healthcare-associated infections are a concern for health service providers, exacerbated by poor delivery of antimicrobials to target sites within the skin. The dermal route is attractive for local an...

Design of Multifunctional Peptide Collaborated and Docetaxel Loaded Lipid Nanoparticles for Antiglioma Therapy.

Glioblastoma multiforme (GBM) is one of the most encountered gliomas of the central nervous system. The chemotherapeutic drugs used in the treatment of GBM suffer from poor blood brain barrier penetra...

Clinical Trials [5280 Associated Clinical Trials listed on BioPortfolio]

Topical Silver Nanoparticles for Microbial Activity

Silver nanoparticles are one of most nanoparticles use nowadays in the research area because it has specific physical and chemical properties, in medical fields silver nanoparticles can in...

Clinical Assessment of Oxiconazole Nitrate Solid Lipid Nanoparticles Loaded Gel

Clinical study for the developed oxiconazole nitrate solid lipid nanoparticles (SLNs) gel system compared to the corresponding marketed product was conducted on 28 patients with different ...

Topical Application of Silver Nanoparticles and Oral Pathogens in Ill Patients

The purpose of this study is to determine whether oral topic silver nanoparticles are effective to reduce potential pathogen microbial loads in mechanical ventilation patients.

Targeted Polymeric Nanoparticles Loaded With Cetuximab and Decorated With Somatostatin Analogue to Colon Cancer

Due to the great toxicity of chemotherapeutic drugs to both the healthy and cancerous area, the efficient targeting could be of great benefit for a patient with advanced or metastatic tumo...

Presence of Nanoparticles in the Amniotic Fluid

The nanoparticles (NP) are defined as particles whose size is no greater than 100 nanometers. However, their impact on health remains little evaluated. Placental transfer of NP has been p...

Medical and Biotech [MESH] Definitions

Nanometer-sized particles that are nanoscale in three dimensions. They include nanocrystaline materials; NANOCAPSULES; METAL NANOPARTICLES; DENDRIMERS, and QUANTUM DOTS. The uses of nanoparticles include DRUG DELIVERY SYSTEMS and cancer targeting and imaging.

Synthesized magnetic particles under 100 nanometers possessing many biomedical applications including DRUG DELIVERY SYSTEMS and CONTRAST AGENTS. The particles are usually coated with a variety of polymeric compounds.

Diamond nanoparticles that exhibit unique biological, thermal, mechanical, and optoelectronic properties. They have important NANOMEDICINE applications including DRUG DELIVERY SYSTEMS; DIAGNOSTIC IMAGING; protein separation; and BIOSENSING TECHNIQUES.

Nanoparticles produced from metals whose uses include biosensors, optics, and catalysts. In biomedical applications the particles frequently involve the noble metals, especially gold and silver.

A layer of protein coating adsorbed by NANOPARTICLES upon entry into PLASMA or other protein-containing biological fluids, which affects how nanoparticles are internalized by cells and cleared from the body.

Advertisement
Quick Search
Advertisement
Advertisement

 


DeepDyve research library

Relevant Topics

Gene Therapy
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...

Bioinformatics
Bioinformatics is the application of computer software and hardware to the management of biological data to create useful information. Computers are used to gather, store, analyze and integrate biological and genetic information which can then be applied...

Gene Expression
The process of gene expression is used by eukaryotes, prokaryotes, and viruses to generate the macromolecular machinery for life. Steps in the gene expression process may be modulated, including the transcription, RNA splicing, translation, and post-tran...


Searches Linking to this Article