Future of Gene Therapy

Advances in gene-based therapies, including siRNA drugs, are expanding treatment options beyond rare genetic disorders to complex conditions like high cholesterol and hypertension.

Future of Therapy: Emerging Approaches

• Shift from Symptom Control to Cure: Unlike conventional drugs that manage disease symptoms, emerging genetic therapies aim to correct molecular mechanisms at their source.
• RNA-Based Therapeutics: Techniques such as small interfering RNA (siRNA) modify gene expression without altering DNA, offering safer and more targeted interventions.
  • siRNA is a short RNA molecule that silences specific genes by degrading their target messenger RNA.
• Long-Lasting Treatment Effects: New therapies, including siRNA-based drugs, can provide sustained benefits from a single injection lasting several months.

About Gene Therapy

• Introduction: Gene therapy involves treating disease by correcting, silencing, or modifying faulty genes or their expression within a patient’s cells.
• How It Works: Genes encoded in DNA are transcribed into RNA and translated into proteins essential for normal body functions; disruptions in this process can cause disease.
• Types of Gene Therapy
• • Somatic gene therapy: Alters non-reproductive cells; effects are not inherited. (e.g., for cancer treatment, inherited disorders like muscular dystrophy)
  • Germline gene therapy: Alters reproductive cells; changes can be passed to offspring (highly restricted/ethical concerns).
• Features of Gene Therapy:
  • Targets the root genetic cause rather than downstream biochemical effects.
  • Can involve gene replacement, gene silencing, or gene editing approaches.
  • Enables high precision in treating conditions caused by specific genetic defects.
• Expanding Scope Beyond Rare Diseases: While initially successful in rare monogenic disorders like spinal muscular atrophy, newer approaches focus on regulating gene activity in common diseases.
• Need for Gene Therapy: Many diseases arise from defective or overactive genes producing harmful proteins, which traditional drugs cannot permanently correct.
  • Gene therapy offers durable solutions, reduces lifelong medication dependence, and improves outcomes where conventional pharmacology has limitations.

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About Small interfering RNA (siRNA) drugs

• Small interfering RNA (siRNA) drugs are gene-silencing therapies that block the expression of specific disease-causing genes by degrading their target messenger RNA (mRNA).
• Key Features:
• • Act at the RNA level, preventing harmful protein production without altering DNA.
  • Highly target-specific, reducing off-target effects.
  • Often delivered to the liver using carriers like GalNAc for efficient uptake.
    • GalNAc (N-acetylgalactosamine) are a type of targeting ligand used to facilitate specific delivery to liver cells (hepatocytes)
  • Provide long-lasting effects with infrequent dosing (months).
  • Effective in treating non-single-gene disorders such as high cholesterol and hypertension.
• Current Examples: FDA-approved siRNA drugs like Inclisiran lower LDL cholesterol by about 50%, while Zilebesiran shows promise as a long-acting treatment for hypertension.

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