What are some successful examples of genetic therapy?

What are some successful examples of genetic therapy?

There have been several remarkable successes in genetic therapy. For instance, the treatment of spinal muscular atrophy (SMA) with Zolgensma has shown significant improvements in patients.

Can you tell me more about Zolgensma?

Zolgensma is a gene therapy that targets the root cause of SMA by delivering a copy of the SMN gene to the patient's cells, helping to produce the SMN protein that is essential for motor neuron function.

Are there other diseases that have benefited from genetic therapy?

Yes, other diseases like certain types of inherited blindness and hemophilia have also seen successful treatments through genetic therapy, improving patients' quality of life.

What are the risks associated with genetic therapy?

While genetic therapies can be life-changing, they may carry risks such as immune reactions, unintended genetic changes, or long-term effects that are still being studied.