DDx

The Future of Gene Therapy and Genetic Diseases


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Peek into the future of gene therapy and its capacity to treat – maybe eliminate – genetic diseases like cancers and hemophilia. Plus, the potential to reverse the effects of aging. 

It’s a future scientists have been working toward for years: How to treat complex health  problems with gene therapy. And researchers have been making progress. Diagnoses once thought to be fatal are now being looked at in a new light.

This is a welcome sight for physicians, caregivers, and – most of all – for the patients living with these genetic diseases. 

One disease that’s impacting lives worldwide is cancer. Nearly 40% of the world’s population will be diagnosed with it at some stage of life.

Typically, cancer treatment takes three forms: chemotherapy, surgery, or radiation therapy. Targeted drug therapies also exist, which work by identifying and attacking cancer cells individually.

But the treatment that many believe has the most potential is immunotherapy.

Immunotherapy uses a patient’s immune system to target and destroy cancerous tumors. And a specific type of immunotherapy known as Chimeric antigen receptor (or CAR) T-cell therapy has particular promise.

Over the last few years, progress with this new class of gene-based treatment has accelerated.

CAR T-cell Therapy is when a patient's own immune cells – the white blood cells called T cells – are genetically altered to target and attack a specific cancer within the body. These cells are first removed from the patient’s blood. Their genes are then altered to produce proteins called CARs, which allow the T cell to better recognize – and attack – specific cancer cells. When the altered immune cells are reintroduced into the patient's bloodstream, these proteins latch onto both healthy and cancerous cells, destroying the cancerous cells while leaving the healthy cells unharmed.

CAR T-cell Therapy has the ability to revolutionize cancer treatment and prevent relapse, as  these cells can potentially continue to attack cancerous cells in a patient’s body for years. But it’s not a solution for everyone. Only about 40% of patients have long-term responses.

But if this therapy achieves what scientists believe it can, chemotherapy could be a thing of the past, and when it comes to the future of gene therapy and genetic diseases, there's reason for optimism.

For more education on gene therapy, visit www.genetherapynetwork.com.

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