What is Gene Therapy and How is it Used?

 

What is Gene Therapy and How is it Used?

Gene therapy is a method used to treat or prevent diseases by altering a person’s genes. It involves introducing new, healthy genes into a patient's body to replace or fix faulty ones. This approach has been explored since the 1990s and shows promise in treating various genetic conditions.

Current Applications of Gene Therapy:

1. Blood Cancers:
   Gene therapy is currently being used to treat certain blood cancers like B-cell acute lymphoblastic leukemia. In this treatment, T-cells (a type of white blood cell) are modified to attack cancerous B-cells. This method, known as CAR-T cell therapy, has been highly effective, with some patients seeing their cancer disappear in just weeks.

2. Blindness (Leber’s Congenital Amaurosis):
   Leber’s congenital amaurosis (LCA) is a rare eye condition that causes vision loss. A gene therapy treatment called voretigene neparvovec can now restore sight by injecting a functional copy of the affected gene directly into the eye cells. This therapy has proven particularly effective in younger patients.

3. Metachromatic Leukodystrophy (MLD):
   MLD is a genetic disorder that damages the nervous system. A new gene therapy has been developed that replaces the faulty gene responsible for the disease. This therapy has shown promising results, particularly in very young patients, offering the possibility of a complete cure.

Potential Future Applications of Gene Therapy:

1. Heart Failure:
   Research is underway to use gene therapy to treat heart failure, a condition where the heart can't pump blood effectively. Scientists are exploring replacing or boosting the SUMO-1 gene in patients to improve heart function.

2. Bone Cancer Treatments:
   Gene therapy is being tested to improve bone grafts used in bone cancer treatment. By activating specific genes, researchers hope to make these grafts more durable and integrate better with the patient’s own bone.

The First Success Story: SCID
The first genetic condition treated using gene therapy was Severe Combined Immunodeficiency (SCID), a condition so severe that patients had to live in complete isolation to avoid infections. This groundbreaking treatment opened the door to further gene therapy research and applications.

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