What is Gene Therapy?

What is Gene Therapy?

Genetic therapies strive to correct errors in your genes by inserting a normal copy of the missing or damaged gene into your cells. Usually, this involves using an artificial vector which transports the new DNA into your cells.

Viral gene transfer: Viruses have the unique capacity to deliver genes into cells, so some are used as vectors to deliver therapeutic genes into patients’ bodies. While this approach can be successful for certain conditions, it may lead to side effects like infection or inflammation.

Plasmid DNA: Circular DNA molecules can be genetically engineered to deliver therapeutic genes into your cells. These are also referred to as plasmids or viral vectors, and have been successfully used to treat diseases like leukemia and lymphoma.

Vaccines: Another method for delivering therapeutic genes into cells is through vaccines. While this treatment option has potential for many conditions, it’s often expensive and difficult to administer.

Liposomes: Delivering genes directly into cells via liposomes has the potential for greater effectiveness than vaccines, though they require more effort and cost. This treatment is currently being tested on people suffering from spinal muscular atrophy – a rare disorder that affects muscles – but may prove more challenging to administer and costlier in the long run.

Gene therapies differ from other medications in that they only need to be given once. This makes them more appealing for people who haven’t responded to other treatments. It is essential to remember, however, that gene therapy is still relatively new and there are still unknowns regarding its risks and advantages.