Blog Post

#021

What is Gene-Editing Therapy?

Gene-editing therapy is a modern medical procedure that involves modifying the DNA of cells to cure genetic diseases, fight cancer, and tackle conditions that were once untreatable. But, how does it work?


Overview:

  • Gene therapy refers to treating diseases by adding, replacing, or blocking genes through mechanisms like viral vectors

  • Genome editing is a specific type of gene editing that involves cutting specific parts of the DNA and modifying it by deleting or changing sequences


Two Main types of Gene-Editing Approaches:

  • Homology-Directed Repair (HDR): Uses a DNA template to precisely fix or insert genes

  • Non-Homologous End Joining (NHEJ): Commonly used when a simple gene needs to be blocked


How do Gene-Editing Therapies Work?

  • Scientists identify which gene or DNA sequences needs to be edited because it causes a disease like sickle cell disease

  • CRISPR, the most common editing tool, and other editing tools have designed molecular scissors to cut specific parts of the DNA

  • Gene-editing tools are delivered into target cells through viral vectors, nanoparticles, or other delivery systems

  • After the cut/editing, the cells naturally repair the DNA using HDR or NHEJ, and the disease-causing gene is now blocked


Clinical Applications:

  • Gene-editing therapies are commonly used for genetic disorders like cystic fibrosis, sickle cell disease, and Huntington’s disease 

  • Gene-editing can be used in engineered immune cells like CAR-T to fight cancer cells more effectively

  • Trials and tests have been going on to fix metabolic pathways or edit genes in the eye or blood to remove inherited blindness or blood disorders


Risks of Gene-Editing Therapies:

  • Immune response: Viral vectors can provoke immune reactions and weaken the immune system

  • Off-target effects: Gene-editing tools can lead to gene disruptions and harmful side effects

  • Long-term monitoring: Gene-editing needs to be monitored to ensure they don’t cause any new mutations or dangerous issues

  • Ethical issues: Germline editing (heritable changes) allows parents to alter their children’s genetic traits’

Gene-editing therapy is super fascinating as it seems unreal. With the ability to make microscopic changes to the DNA with technologies like CRISPR and viral vectors, scientists are continuing to develop treatments to fix genetic disorders and diseases. However, there are risks involved that can negatively affect society like off-target effects or germline editing. It is important to understand that gene-editing therapy still requires trials and safety measures to ensure it has the potential to offer cures.


Gene-Editing Therapies