Question

Discuss the two main therapeutic avenues to treat lysosome storage diseases. How does one strategy trick the cell into taking up a drug and deliver it to the lysosome?

Answer 1

lysosome are the particular interest for the design and deliver of pH dependent pro- drugs, enhancing selectivity and enhancing strategies to inhibit drug degradation inside the cells. there is the great potential to bring intracellular drug delivery and distribution using nano - therapeutic approach to target lysosome for therapeutic intervention. lysosomal targetting strategy involve two contrasting facets. one aspect is to directly targeted therapeutic to the lysosome through receptor mediated endocytosis and the other facet involve strategies which ensure escape from the lysosome in order to prevent to prevent their degradation, so that therapeutic may remain intact and available in the cytosol for their further action. it provide the unique opportunities to explore novel treatment strategies and design future drugs for effective treatment of lysosome related disease , especially lysosomal disorder disease (LSD), cancer inflammatory, neuro degenerative condition ( parkinson's , alzheimer's, and hutington's disease) and autoimmune disease . In this review, we illustrate the fundamental of membrane trafficking, subcellular organization, strategies to target lysosomes and its implication for the advance design of efficient drug delivery vector for safe and effective therapy.