Editorial for Special Issue on Gene Therapy of Rare Diseases.

WHY IT MATTERS

This editorial highlights the growing momentum in gene therapy development for rare diseases, which means more treatment options may become available for conditions that previously had few or no approved therapies.

This is an editorial article that introduces a special collection of scientific papers about gene therapy for rare diseases. Gene therapy is a treatment that fixes or replaces faulty genes that cause disease. The editorial discusses how gene therapy is becoming an important treatment option for rare diseases that affect small numbers of people.

Editorial for Special Issue on Gene Therapy of Rare Diseases. Abstract: A rare disease is a condition that affects only a small portion of the population [...]. Authors: Brakebusch Journal: Cells MeSH: Rare Diseases, Humans, Genetic Therapy