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1. Explore - Genetic Diseases: Challenges and Solutions

1.6 Emerging Solutions

Recent advances in biotechnology offer promising solutions to genetic diseases:

  • Gene therapy uses viral vectors to deliver healthy copies of genes into affected cells.
  • CRISPR/Cas9 gene editing allows scientists to precisely modify DNA, correcting mutations at their source.
  • RNA-based therapies and cell therapies aim to restore normal protein production or replace damaged cells.

Some of these approaches have already reached clinical use, demonstrating how basic genetic knowledge can lead to real medical breakthroughs.