1.5 Treatment Challenges

Traditional treatments for genetic diseases usually focus on managing symptoms rather than correcting the underlying genetic cause. While these approaches can improve quality of life, they have important limitations:

  • The mutated gene itself remains unchanged.
  • Damage to tissues and organs may be irreversible.
  • Delivering treatments to specific cells, such as those protected by biological barriers, remains difficult.

These challenges highlight the need for new therapeutic strategies.