Researchers Develop Breakthrough mRNA Delivery Technique
Table of Contents
- 1. Researchers Develop Breakthrough mRNA Delivery Technique
- 2. What are the potential benefits of using EVs for mRNA delivery compared to current lipid nanoparticle (LNP) based systems?
- 3. Australian Researchers Advance mRNA Delivery Technique
- 4. The Challenge of mRNA Therapeutics
- 5. breakthrough: Utilizing Extracellular vesicles (EVs)
- 6. How the New Technique Works: A Step-by-Step Breakdown
- 7. Advantages Over Existing mRNA Delivery Systems
- 8. Applications and Potential Therapeutic Areas
- 9. Real-World Implications & early Trials
- 10. The Role of Exons, Introns, and the Coding Sequence
Sydney, Aug. 5 (Xinhua) — Researchers In Australia Have Developed A Technique That Greatly Improves Targeted mRNA Delivery To Cells, Paving The Way For Advanced therapies Beyond Vaccines.
the Method marks A Major Advance In Delivering mRNA Precisely To Cells, Boosting Treatment Efficacy Adn Minimizing Off-Target Effects, Or side Effects, For Future Therapies. This data Comes According To A Statement Released Tuesday By monash University In Australia’s Melbourne.
Researchers Developed A Versatile Method That Captures And Attaches Antibodies In optimal Orientation To mRNA-Loaded “Lipid Nanoparticles” — Tiny Fat-based Spheres That protect And Deliver mRNA Drugs To Their Destination. Improving Effectiveness And Reducing Side Effects By Targeting Delivery Is A Key Benefit.
This Innovation, Led By Monash Institute Of Pharmaceutical Sciences (MIPS), Has Increased The Binding of mRNA To Target Cells Eightfold Compared To Conventional Approaches. It Also Significantly Reduced Unintended Delivery To Other Cells.
“This Level Of Control Opens Up New Possibilities For Developing mRNA Medicines With Far Greater Specificity,” Said MIPS PhD Moore Zhe Chen, Co-Lead Author Of The Study Published In Nature Nanotechnology.
Co-Lead Author, MIPS Associate Professor Angus Johnston, Highlighted That Efficient And Precise mRNA Delivery Is Critical To Advance mRNA Medicines Beyond Their Current Use as Vaccines. Building On The Proven Success Of Lipid Nanoparticles In COVID-19 vaccines Is The Goal.
Unlike Existing Techniques That Require Antibody Modification — Potentially Reducing Their Efficacy And Limiting Their Submission To Vaccines — The MIPS Method allows Antibodies To Be Used Unaltered.Johnston Explained This Vital distinction.
“In This Study, We Used Powerful Imaging Techniques To Develop A simple Antibody Capture System That Requires No Modification Of The Antibody, and Ensures The Antibodies Are Attached Onto Lipid nanoparticles In An Orientation That Increases Binding To Target Cells,” He Said.
“This Is vital For Developing New mRNA Medicines Beyond Vaccines.”
Preclinical Studies Confirmed The Method’s Effective Delivery Of mRNA To T Cells In Mice, With Minimal Off-Target Effects On Other immune Cells, According To The researchers.
The MIPS Team Is Advancing This Platform To enable Precise mRNA Delivery For Targeted Treatments In Cancer, Genetic disorders, And Autoimmune Diseases, Where Targeted Therapies Could Dramatically Improve Outcomes.
Disclaimer: This article discusses medical research. It is indeed not intended to provide medical advice. Please consult with a qualified healthcare professional for any health concerns or before making any decisions related to your health or treatment.
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What are the potential benefits of using EVs for mRNA delivery compared to current lipid nanoparticle (LNP) based systems?
Australian Researchers Advance mRNA Delivery Technique
The Challenge of mRNA Therapeutics
Messenger RNA (mRNA) therapeutics hold immense promise for treating a wide range of diseases,from infectious diseases like COVID-19 to genetic disorders and even cancer. However, a meaningful hurdle in realizing this potential lies in efficient and safe delivery of mRNA into cells. Naked mRNA is rapidly degraded by enzymes in the body and struggles to enter cells effectively. current delivery systems, primarily lipid nanoparticles (LNPs), while successful, aren’t without limitations – potential toxicity, off-target effects, and manufacturing complexities. Australian researchers are now making strides to overcome these challenges with a novel approach to mRNA delivery.
breakthrough: Utilizing Extracellular vesicles (EVs)
Researchers at the University of Queensland and the Australian National University have announced a significant advancement in mRNA delivery, focusing on extracellular vesicles (EVs). EVs are naturally occurring nanoscale vesicles secreted by cells, acting as natural messengers within the body. They possess inherent biocompatibility and the ability to cross biological barriers, making them ideal candidates for drug delivery.
This new technique doesn’t involve creating EVs, but rather re-engineering them. The team has developed a method to load EVs with mRNA and enhance their targeting capabilities. This is a departure from traditional LNP-based systems and offers several potential advantages.
How the New Technique Works: A Step-by-Step Breakdown
The process involves several key steps:
- EV Isolation: EVs are isolated from cell cultures – specifically, cells chosen for their natural ability to produce EVs with favorable characteristics.
- mRNA Encapsulation: A proprietary method is used to efficiently encapsulate mRNA within the EVs. This process protects the mRNA from degradation and enhances its cellular uptake. Details remain closely guarded pending patent applications.
- Surface Modification: The surface of the EVs is modified with targeting ligands – molecules that bind to specific receptors on target cells. This ensures the EVs deliver their mRNA payload precisely where it’s needed, minimizing off-target effects. Researchers are exploring various ligands for different tissue types and disease states.
- Enhanced Cellular Uptake: The modified EVs demonstrate considerably improved cellular uptake compared to unmodified EVs or mRNA delivered via conventional methods.
Advantages Over Existing mRNA Delivery Systems
This Australian innovation presents several compelling advantages over current LNP technology:
Enhanced Biocompatibility: EVs are naturally derived, reducing the risk of immune responses and toxicity associated with synthetic materials like lipids.
Improved Targeting: Surface modification allows for precise targeting of specific cells and tissues, maximizing therapeutic efficacy and minimizing side effects. This is crucial for personalized medicine approaches.
Natural Biodegradability: EVs are naturally broken down by the body, eliminating concerns about long-term accumulation of delivery materials.
Potential for Scalability: While still in early stages, researchers believe the EV production process can be scaled up for commercial manufacturing.
Reduced Off-Target Effects: The targeted delivery minimizes exposure of healthy tissues to the mRNA therapeutic.
Applications and Potential Therapeutic Areas
The potential applications of this advanced mRNA delivery technique are vast.Researchers are initially focusing on:
Cancer Immunotherapy: Delivering mRNA encoding tumor-specific antigens to stimulate an immune response against cancer cells.
Genetic Disease Treatment: Correcting genetic defects by delivering mRNA encoding functional proteins. This is especially promising for diseases like cystic fibrosis and muscular dystrophy.
Vaccine Development: Creating more effective and targeted vaccines against infectious diseases. The technology could perhaps improve vaccine efficacy and reduce the need for booster shots.
Regenerative Medicine: Delivering mRNA to promote tissue repair and regeneration.
Real-World Implications & early Trials
While still in the pre-clinical phase, initial in vitro and in vivo* studies have shown promising results. Researchers have demonstrated successful mRNA delivery and protein expression in various cell types and animal models.
A small-scale pilot study is planned for late 2025, focusing on delivering mRNA-based cancer immunotherapy to patients with advanced melanoma. This trial will primarily assess the safety and feasibility of the EV-based delivery system. The team is collaborating with several pharmaceutical companies to accelerate the development and commercialization of this technology.