A Historical Look at Gene-Based Therapies

Gene replacement therapy takes a different path to potential treatment because it addresses the root cause of the disease. Gene-based therapies (broadly referred to as “gene therapy”), however, are not a new concept. Scientists have been exploring gene therapy for decades as a way to treat genetic illness. Take a look at this timeline of major milestones in gene-based therapies.

A timeline of progress in gene-based therapies

  • Pea plants

    Working with pea plants, a scientist named Gregor Mendel discovers the fundamentals of heredity and how genetic information is passed from parent to child.

  • The term “gene” is coined.

  • DNA Molecule

    James Watson, Francis Crick, and Maurice Wilkins characterize the structure of DNA. They were awarded the Nobel Prize in Physiology or Medicine in 1962 for this discovery.

  • Waclaw Szybalski demonstrates the first successful gene repair in mammalian cells.

  • The adeno-associated virus is discovered (microscope)

    The adeno-associated virus (AAV) is discovered. It will eventually be modified by scientists and used as a vector to deliver a new, working gene to cells.

  • Rogers and Pfuderer demonstrate that virus-mediated gene transfer is possible.

  • The concept of gene therapy is first considered as a treatment for genetic diseases.

  • The first direct human gene therapy trial took place, and while the trial was unsuccessful, there were many important learnings.

  • Scientists embark on a 15-year odyssey that culminated in successfully mapping every piece of DNA in the human body.

    Mapping DNA
  • A gene-based therapy is shown to be able to correct a genetic defect in human cells.

  • In-human gene transfer

    Rosenberg conducts the first in-human gene transfer.

  • The FDA, for the first time, approves a human gene therapy trial. Gene therapy is used to treat a 4-year-old girl and a 9-year-old girl with a genetic disease.

    During this time, an 18-year-old boy undergoing gene therapy in a clinical trial passed away. This temporarily slows down gene therapy research so scientists can understand what happened. Scientists continue in their pursuit of a way to safely treat genetic diseases.

  • China approves the first gene therapy in the world for head and neck cancers.

  • Scientists show that an AAV vector has the potential to cross the blood-brain barrier in the body. This is a major advancement toward the treatment of genetic diseases with gene replacement therapy.

    A brain
  • The first gene therapy is approved by the European Medicines Agency to treat a rare genetic disease.

  • The FDA approves the first CAR-T cell therapy in the United States, which is used to treat acute lymphoblastic leukemia. By the end of 2017, the FDA approves another gene therapy for an inherited form of vision loss.

  • More than 2,600 gene therapy clinical trials completed, ongoing, or approved worldwide. A major medical journal declares, “Gene Therapy Comes of Age.”

Check your understanding of gene replacement therapy


When was the adeno-associated virus (AAV) first discovered?

The adeno-associated virus (AAV) was first discovered in 1965. This advancement, more than 50 years ago, has made a significant contribution to gene replacement therapy today.