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May 23, 2024 : RNA-based therapeutics hold immense promise for revolutionizing the treatment of various diseases. These therapies harness the power of ribonucleic acid (RNA) to modulate gene expression, offering a novel approach to tackling complex medical challenges. However, unlocking the full potential of this technology requires the development of robust frameworks for drug design, delivery, and clinical application.
Researchers are actively exploring various RNA modalities, each with its unique therapeutic mechanism. These modalities include messenger RNA (mRNA) for protein replacement, small interfering RNA (siRNA) for gene silencing, and antisense RNA (asRNA) for targeted RNA degradation. Each modality offers distinct advantages but also presents its own set of challenges.
The development of efficient and safe delivery systems is crucial for advancing RNA-based therapies. The inherent properties of RNA molecules, such as their susceptibility to enzymatic degradation, pose a significant hurdle. Effective delivery systems are necessary to protect RNA therapeutics from degradation and ensure their targeted delivery to specific tissues or cells within the body.
Lipid nanoparticles (LNPs) have emerged as a promising solution for RNA delivery. These biocompatible carriers encapsulate RNA molecules, shielding them from degradation and facilitating cellular uptake. However, further research is needed to optimize LNPs for improved targeting efficiency and reduced potential side effects.
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