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Gene-targeted therapies (GTTs) offer a promising approach for treating numerous monogenic diseases. This review summarizes current GTT types and their scientific advancements, aiding rare disease therapy development.

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Area of Science:

  • Genetics
  • Therapeutics
  • Rare Diseases

Background:

  • Gene-targeted therapies (GTTs) represent a significant advancement in medicine.
  • These therapies are applicable to a wide range of monogenic diseases.
  • The development of GTTs has major implications for rare disease treatment.

Purpose of the Study:

  • To provide a concise summary of the main types of GTTs.
  • To offer an overview of the current scientific landscape of GTTs.
  • To serve as an introductory primer for related articles in this special issue.

Main Methods:

  • Review of existing literature on gene-targeted therapies.
  • Categorization of primary GTT types.
  • Synthesis of current scientific data and trends.

Main Results:

  • Identification and categorization of key GTT platforms.
  • Summary of the current state of scientific development in GTTs.
  • Establishment of a foundational understanding for further research.

Conclusions:

  • GTTs are versatile platforms with the potential to treat numerous monogenic conditions.
  • The rapid progress in GTTs is transforming rare disease therapy development.
  • This article provides essential context for understanding the evolving field of GTTs.