Explain about RNA therapeutics and its applications with examples.
Show Hint
RNA therapeutics have revolutionized the treatment of previously untreatable diseases, with mRNA vaccines and RNAi therapies leading the charge in personalized medicine.
RNA therapeutics involves the use of RNA molecules to treat diseases by either modifying or replacing the defective or missing RNA in a patient’s cells. This emerging field holds significant promise for treating a wide range of diseases, including genetic disorders, cancers, and viral infections.
Step 1: Types of RNA Therapeutics:
1. mRNA Therapeutics: Messenger RNA (mRN is used to instruct cells to produce proteins that can treat or prevent disease. mRNA vaccines, for example, are designed to provoke an immune response against a specific pathogen.
2. RNA Interference (RNAi): RNAi therapeutics aim to silence or reduce the expression of specific genes by using small RNA molecules, such as small interfering RNAs (siRNAs) or microRNAs, to target and degrade messenger RNAs (mRNAs) that code for disease-causing proteins.
3. Antisense Oligonucleotides (ASOs): These are short RNA molecules that bind to specific mRNA sequences, preventing the production of harmful proteins or correcting splicing defects in genetic diseases.
Step 2: Applications of RNA Therapeutics:
1. mRNA Vaccines: The development of COVID-19 vaccines such as Pfizer-BioNTech and Moderna, which use mRNA technology to instruct cells to produce the spike protein of SARS-CoV-2 and trigger an immune response.
2. Gene Silencing in Genetic Diseases: RNAi-based therapies, such as Onpattro (patisiran), are used to treat hereditary transthyretin amyloidosis by silencing the gene responsible for producing the defective protein.
3. Antisense Oligonucleotide Therapy: Spinraza (nusinersen), used to treat spinal muscular atrophy, works by correcting the splicing of the SMN2 gene, leading to the production of functional SMN protein.
