Human Gene Therapy

The concept of gene therapy is centered on addressing genetic disorders at their source. For example, if a mutation in a specific gene leads to the production of a dysfunctional protein—often resulting in a genetic disease—gene therapy aims to introduce a healthy copy of that gene. This healthy gene can then produce a functional protein, effectively compensating for the defective one.

This approach, known as gene replacement therapy, is being used to treat various inherited conditions, including hereditary retinal diseases, by restoring normal cellular function at the genetic level.

  • Heterogeneity
  • Heterogeneous gene-expression

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