What is gene therapy?
What is gene therapy?Gene therapy is a way to treat a disease by correcting the abnormal genetic problem that causes it. This one-time treatment involves fixing errors in our DNA: the instructions that tell our bodies how to work. By changing these genetic instructions, gene therapy can restore important functions in the body.
What diseases do we treat with gene therapy?
We offer gene therapy at NewYork-Presbyterian/Columbia to treat sickle cell disease in patients age 12 and older with a history of vaso-occlusive events and no matched donor for a bone marrow transplant. The treatments include the drugs exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia).
Casgevy is also a gene therapy for people with beta-thalassemia who are dependent on blood transfusions.
Investigators at NewYork-Presbyterian are also evaluating gene therapy for:
- Cystic fibrosis
- Genetic hearing loss in children
- Amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease)
- Parkinson's disease
Gene therapy approaches
Gene therapy may be accomplished by:
- Replacing faulty genes with healthy ones
- Adding new genes to help fight illness
- Turning off genes that cause disease
More recently, scientists have created gene therapy approaches using a novel technology called CRISPR to edit a patient's faulty genes. Casgevy became the first therapy approved for use in the United States that uses this approach.
How does gene therapy work?
How does gene therapy work?Different gene therapies work in different ways. Casgevy, for example, is made from a patient's own blood stem cells that have been removed and modified.
- In a lab, CRISPR is used to make a precise edit in a gene that controls the production of fetal hemoglobin, a type of hemoglobin we all produce until shortly after birth. People with sickle cell disease inherited a gene from both parents that has instructions for making abnormal hemoglobin. The abnormal hemoglobin causes red blood cells to sickle.
- After giving Casgevy back to the patient, the cells take hold in the bone marrow and begin forming red blood cells that make fetal hemoglobin.
- When the red blood cells produce a certain level of fetal hemoglobin, the cells no longer become sickled or sticky.
What to expect during gene therapy
What to expect during gene therapyIf you are preparing to have gene therapy, your doctor will let you know exactly what to expect.
Here is how patients receiving Casgevy gene therapy for sickle cell disease are typically treated:
- The first step is collecting stem cells so that they can be edited in the laboratory with CRISPR before they are returned to the patient.
- The patient receives intensive chemotherapy in the hospital to eliminate some stem cells and make space in the bone marrow for the new blood-forming cells. This part of the treatment requires a lengthy, weeks-long hospital stay while the patient's immune system recovers.
- The patient receives Casgevy through an infusion and starts making red blood cells that contain fetal hemoglobin.
- The patient stays in the hospital until their care team feels they are ready to go home.
- Patients who suffered daily no longer experience pain crises or require hospitalizations.
- Unlike other treatments, patients who receive gene therapy for sickle cell disease do not need to come back for repeat treatments.
Side effects of gene therapy
Side effects of gene therapyGene therapy is a lengthy, intense process. Its side effects are similar to those observed during the chemotherapy portion of bone marrow transplantation. The chemotherapy can have side effects such as mouth sores, nausea, vomiting, diarrhea, hair loss, and long-term effects on fertility. Your doctor will discuss possible side effects with you.
Why choose us
Why choose usNewYork-Presbyterian/Columbia has been a world leader in gene therapy. We are one of the few centers that had early access to gene therapy for people with sickle cell disease. In fact, one of our patients was just the third in the world to get a CRISPR-engineered treatment for sickle cell disease.
Contact us today to make an appointment and learn more about our services and what we can offer to help you or your loved one start living a healthier, more comfortable life.
