Is it Possible to Treat Blindness with Lipid Nanoparticle Technology?
Latest studies in precision medicine have shown progress toward managing human health and overall way of living. One such study comes from Oregon Science University College of Pharmacy, according to which genetically acquired blindness can now be treated through the assistance of lipid nanoparticles (LNPs).
Researchers used LNPs and messenger RNA (mRNA) in animal models as a new approach to gene therapy in the treatment of inherited retinal degeneration (IRD).
IRDs are a group of inherited genetic disorders that affect the retina, leading to progressive vision loss and blindness. There are multiple types of IRDs, such as color blindness, glaucoma, and cataract, caused by mutations in different genes.
LNPs can be used to deliver therapeutic genes that are normal copies of the mutated genes to the retinal cells that will treat IRDs. Furthermore, the LNPs enter the neural retina and deliver mRNA to the photoreceptor cells, which are responsible for eye vision.
Once delivered, these therapeutic genes can correct the genetic defect, slowing or stopping the progression of the disease.
With the rise in the incidence of chronic diseases, such as IRD and cancer, numerous clinical trials, advancements in technology, and extensive research on the genetic makeup of humans, the global cell and gene therapy market is expected to be on the rise.
As per the report by BIS Research, the global cell and gene therapy market was valued at $2.59 billion in 2020, and it is expected to grow at a CAGR of 33.82% and reach a value of $25 billion in 2027.
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Understanding the Process
Earlier, the role of LNPs was limited to delivering drugs such as COVID-19 vaccines and treating eye diseases such as age-related macular degeneration (AMD) and retinitis pigmentosa (RP).
Now LNPs can treat defective retinas with their newly found ability to reach the back of the retina. As LNPs protect the drug from degradation, it helps it to reach the target site more effectively.
Gaurav Sahay, one of the lead researchers, said, “We identified a novel set of peptides that can reach the back of the eye. We used these peptides to act as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye.”
Interestingly, another lead researcher of the study, Marco Herrera-Barrera, adds, “The peptides that we have discovered can be used as targeting ligands directly conjugated to silencing RNAs, small molecules for therapeutics or as imaging probes.”
LNPs, with a coating of peptides, deliver strands of mRNA to photoreceptors that will produce protein. This protein would allow the retina to see images and send messages to the brain through the optic nerve. This mRNA-triggered protein can effectively treat hereditary blindness by treating vision-degrading gene mutations.
Researchers at Oregon Science University Replacing Adeno-Associated Virus (AAV) with LNPs
The main objective of the research is to tackle the challenges associated with the primary means of delivery of therapeutic genes that are currently in use, such as AAV.
AAV is a small, non-pathogenic virus that has been used as a vector in gene therapy to deliver therapeutic genes to cells that corrects genetic defects. AAV can integrate its genetic material into a specific location on chromosome 19 without disrupting any important genes.
Moreover, AAV has a low level of host immunity, which means that the body’s immune system is less likely to attack the virus and the therapeutic genes it is delivering.
AAV has been used in gene therapy for a wide range of diseases, such as IRDs, hemophilia, and Parkinson’s disease.
According to Gaurav Sahay, AAV has limited packaging capacity when compared to LNPs. The virus has been unsuccessful in delivering large gene-editing machinery and has also caused treatment failures due to off-track gene-editing that triggered immune responses from the body cells. He further added, “We are hoping to use what we’ve learned so far about LNPs to develop an improved gene editor delivery system.”
Researchers are now replacing AAVs with LNPs as they are more flexible in size and can effectively deliver mRNA, which helps in targeted gene editing.
In the experiment on animal models, the scientists devised green fluorescent protein to prove that LNPs actively reached the photoreceptors at the back of the retina. This new discovery can lead to major developments in gene therapy, which can help people with vision impairment.
Conclusion
LNPs have been shown to be an effective delivery method for therapeutic drugs to retinal cells in laboratory studies.
The LNPs are able to cross the blood-retinal barrier and target specific cells in the retina, allowing for a more targeted and efficient delivery of drugs compared to traditional methods. Thus, LNP technology offers a promising future for the effective treatment of inherited blindness and IRDs.
However, it’s important to note that these studies are still in the experimental phase, and more research is needed to determine the safety and efficacy of this method in humans.
Further developments, such as retinas engineered in labs, by scientists at the University of Wisconsin, with future potential for human transplantation, may also help treat human vision impairment and various other retinal disorders.
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