Recent 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 with lipid nanoparticles (LNPs).
Researchers used LNPs and messenger RNA (mRNA) in animal models as a new approach to gene therapy in treating inherited retinal degeneration (IRD).
IRDs are inherited genetic disorders affecting the retina, leading to progressive vision loss and blindness. There are multiple types of IRDs, such as color blindness, glaucoma, and cataracts, caused by mutations in different genes.
LNPs can deliver therapeutic genes that are regular copies of the mutated genes to the retinal cells that treat IRDs. Furthermore, the LNPs enter the neural retina and deliver mRNA to the photoreceptor cells 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.
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 get to 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 as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye."
Interestingly, another study's lead researcher, 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.
AAV is a small, non-pathogenic virus used as a vector in gene therapy to deliver therapeutic genes to cells that correct genetic defects. AAV can integrate its genetic material into a specific location on chromosome 19 without disrupting any essential 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 various diseases, such as IRDs, hemophilia, and Parkinson's. According to Gaurav Sahay, AAV has limited packaging capacity compared to LNPs. The virus has been unsuccessful in delivering large gene-editing machinery. It has also caused treatment failures due to off-track gene editing that triggered immune responses from the body cells. He added, "We hope 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 discovery can lead to significant developments in gene therapy, which can help people with vision impairment.
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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