Small interfering RNA (siRNA) and messenger RNA (mRNA) therapeutics represent transformative approaches in precision pharmacology, enabling targeted gene silencing and protein expression, respectively. This review provides a comprehensive analysis of their molecular mechanisms, delivery strategies, clinical applications, and future directions. By leveraging RNA interference for siRNA and translational machinery for mRNA, these therapeutics address previously untreatable diseases, including genetic disorders, cancers, and infectious diseases. Advances in nanoparticle- based delivery systems, such as lipid nanoparticles, have overcome historical barriers like RNA instability and immune activation. Current clinical applications, including FDA-approved therapies, highlight their efficacy, while ongoing challenges, such as off-target effects and scalable production, are discussed. Future directions emphasize personalized medicine, combination therapies, and novel delivery platforms to enhance therapeutic precision and accessibility.
Ritu Dahiya, A. Singh, Pooja Mathur et al.· Current Gene Therapy· 0 citations
Alzheimer's and Parkinson's diseases are devastating brain disorders. The complex pathophysiology of the diseases and the lack of effective treatments have left them almost unexplored and untreatable. One potential approach to PD and AD therapy development is through exosomes, a delivery system that can be translated from innovative delivery techniques into clinical use. These exosome-based therapeutics will require thorough testing, research-driven refinement of engineering methods, and collaboration among scientists, clinicians, and industry to develop exosome therapeutics for clinical use. This review explores the biological properties of exosomes, recent engineering advances to improve their therapeutic potential, and new methods to leverage their versatility for selective delivery of remedial agents to the brain. In addition, preclinical evidence demonstrates that exosomes can modulate amyloid-β aggregation, α-synuclein pathology, neuroinflammation, and mitochondrial dysfunction. Yet difficulties related to mass production, maintaining quality, and obtaining regulatory approvals to bring them into clinical practice remain significant limiting factors. The authors point out the therapeutic advantages and drawbacks of exosome-based drug delivery systems. Besides, it provides a roadmap for harnessing these methods effectively as medical interventions for Alzheimer's and Parkinson's disorders, thereby promoting more studies in the area. Finally, we outline a conceptual model for translating novel exosome-based delivery methods into clinically applicable treatment modalities for Alzheimer's and Parkinson's diseases, thereby encouraging continued exploration in this promising field of research.
M. Abubakar, Ritu Dahiya, Lokesh Nama et al.· CNS and Neurological Disorde...· 0 citations
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