siRNA Drug Delivery for Cystic Fibrosis: A Review

Cystic Fibrosis (CF) is a genetic condition that impacts life expectancy, caused by mutations in CFTR gene, which interferes with ion transport across epithelial membranes. This condition primes to manufacture of thick, dehydrated mucus that can block airways &ducts, especially in the lungs &pancreas, resulting in ongoing infections and gradual tissue harm. Even with the progress made in pharmaceutical treatments like CFTR modulators, finding a definitive cure continues to be a challenge. Nonetheless, the clinical promise of siRNA faces obstacles such as swift degradation by nucleases, inadequate membrane permeability, restricted mucus penetration, and challenges in effective intracellular delivery. Improved delivery methods are essential to protect siRNA and ensure accurate targeting to the areas in need. This review discoversprobable of siRNA-based DDS in treatment of cystic fibrosis, highlighting the role of non-viral carriers like lipid-based nanoparticles, cationic polymers, dendrimers&peptide-mediated vectors. There is a strong focus on bio-inspired methodologies, including exosomes and mucus-penetrating particles. Special attention is given to tackling the unique challenges posed by cystic fibrosis, including changes in mucus composition and the presence of inflammation. Recent advancements in preclinical research, innovative inhalation formulations, and initial findings from clinical trials are examined to offer a comprehensive understanding of the current developments. The research also explores how siRNA therapies can be combined with existing CFTR modulators to enhance their effectiveness. Ultimately, it explores future paths focused on personalized delivery methods, cutting-edge nanotechnology, and navigating regulatory challenges. Continuous progress in siRNA delivery presents a promising opportunity for a transformative therapy that could change the course of cystic fibrosis.