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Gene Therapy

Gene Therapy

Gene therapy is an experimental technique for treating disease by altering the patient's genetic material. Most often, gene therapy works by introducing a healthy copy of a defective gene into the patient's cells. In recent years there have been rapid advancements in techniques that make it easier than ever to edit the human genome. Genome editing techniques, such as CRISPR/Cas9 allow scientists to edit the genome, by removing, replacing, or adding to parts of the DNA sequence.

Gene Therapy
Images and animations are courtesy of the National Human Genome Research Institute's Talking Glossary (

Materials for Gene Therapy


Genome Editing with CRISPR-Cas9

Description: This animation depicts the CRISPR-Cas9 method for genome editing, a powerful new technology with many applications in biomedical research, including the potential to treat human genetic disease.


Challenges in Gene Therapy

Description: An article exploring some of the major challenges in gene therapy.

Gene Therapy

Description: A rich source of interactive tutorials, games, and articles on gene therapy.

Gene Therapy

Description: A short introduction to gene therapy and listing of conditions for which gene therapy is being investigated

Gene Therapys First Out-and-Out Cure Is Here

Description: A treatment now pending approval in Europe will be the first commercial gene therapy to provide an outright cure for severe combined immune deficiency. This rare disease leaves newborns with almost no defense against viruses, bacteria, or fungi.

Genome Editing

Description: Learn more about how genome editing works, how it is being used, ethical concerns, and what is happening right now.

Treating the Bubble Babies: Gene Therapy In Use

Description: Some children with severe combined immunodeficiency (SCID), a genetic disorder characterized by a reduced number of immune cells, have been treated using gene therapy.

What is CRISPR-Cas9

Description: Facts about CRISPR-Cas9, a genome editing tool that is creating a buzz in the science world.

What Is Gene Therapy?

Description: Gene therapy is when DNA is introduced into a patient to treat a genetic disease. The new DNA usually contains a functioning gene to correct the effects of a disease-causing mutation.

What is genome editing and how does it work?

Description: New scientific techniques are constantly being developed, and some of them have the potential to make a large impact on future research and have possible applications for health. One technique showing great promise is CRISPR-Cas9 – which can be used as a genome-editing tool.


Space Doctor

Description: Play this game to treat sick aliens at the Extraterrestrial Gene Therapy Lab.


Interactive Tutorials

Teacher Resources

Gene Therapy - Molecular Bandage (Lesson Plans/Lesson Activities)

Description: A module with six engaging lesson plans and activities on gene therapy.

Genome Editing and CRISPR Lesson Plan (Lesson Plans/Lesson Activities)

Description: Through this lesson plan you will explore with your students how advances in our ability to change genomes may impact individuals and society.


Watch KQED Hack DNA With a DIY Kit

Description: CRISPR gene-editing technology allows scientists to make changes to specific cells. It has created a lot of excitement in the scientific world. KQED teaches us a bit more by actually doing it using a CRISPR kit they purchased.