Gene Therapy

Gene therapy is a unique medical approach that involves modifying a person's genetic material to treat or prevent diseases. Gene therapy can replace faulty genes with healthy ones, introduce new genes into the body where they may be missing, or modify how much a gene is expressed in the body. By targeting specific genes associated with conditions, gene therapy offers the potential to provide long-term and potentially curative treatments. This innovative field holds promise for personalised and precise medical interventions.

Gene therapy falls into two main categories, ex vivo and in vivo. Ex vivo gene therapy involves removing cells from a patient, introducing the desired genetic material, then reintroducing the cells to the patient through a vector. In vivo gene therapy uses a technique where a vector carrying the desired genetic material is introduced directly into the patient. 

In the past, SMA has posed significant challenges in treatment. However, recent advancements have led to the development of innovative treatments like gene replacement therapy. This treatment involves a one-time delivery of a functional copy of the SMN gene using a vector, which allows the body to produce the missing SMN protein and support muscle function.

Early intervention using gene therapies can significantly alter the natural course of the condition and improve motor function. Ongoing research and clinical trials continue to explore further refinements and applications of gene therapy in addressing SMA and other genetic conditions, fostering optimism for the future of those affected by them.

Gene Therapy Resources

Here are some fantastic resources for you to download about gene therapy and SMA.

Gene Therapy for SMA

Understanding SMA

Zolgensma in Australia