siRNA: A NOVEL THERAPEUTIC APPROACH

siRNA (short for short interfering RNA/silencing RNA) are one among several well-known types of RNA like the miRNA (micro RNA) and the piRNA (piwi interacting RNAs). They play a vital role in RNAi, short for RNA interference, in gene silencing by inhibiting translation of mRNA. These are 20-30 bp long and often emerge exogenously due to viral elements or transposons.[1] In recent years, synthetically engineered siRNA sequences with complementarity to disease causing mRNA have been used in different delivery systems like liposomes and polymeric nanoparticles to silence the expression of diseased genes, hence paving the way to numerous therapeutic approaches in cancer treatment as well as degenerative diseases.

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siRNA in treating neurodegenerative diseases 

 

1. Alzheimer’s disease (AD)

Endogenous Tau inhibition can stop AD-related neural problems. RNAi-based amyloid precursor protein (APP) inhibition can be used to reduce the accumulation of beta-amyloid plaques in the brain. Delivery approaches have varied from sending naked siRNA to using lentiviral vectors. Effects of doing this include inhibition of plaque producing proteins, axonal regeneration, improvements in synaptic activity, etc. 

 

2. Huntington’s disease (HD)

Although there is no specific treatment for the disease in the market, siRNA therapies that target the htt mRNA may offer an essential therapeutic approach for treating HD. In adulthood, turning off aberrant htt expression can minimise HD symptoms. 

 

3. Multiple Sclerosis (MS)

Several targets have been focused on to use siRNA to prevent the progression of MS, LINGO-1 (suppresses oligodendrocyte differentiation and myelination), caspase-2 being few of the many targets examined. Chitosan particles and naked siRNA were used as delivery methods respectively, resulting in better motor functions in the former and inhibition of nerve loss in the latter, among several other silver linings.

 

Due to its precise targeting and prolonged effect, siRNA can overcome some of the drawbacks of traditional drugs. For degenerative disorders, particularly neurological diseases, where a longer period of activity is required, siRNA may be an effective treatment option. Studies on the effectiveness of siRNA therapies for degenerative illnesses as well as their transport to the site of action using polymeric, lipid-based, and viral vehicles have been conducted in a number of ways. More research should be done, nevertheless, on the negative consequences of siRNA as well as on secure siRNA delivery methods.