can gene therapy treat inherited diseases
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.n the future, this technique may allow doctors to treat a disorder by inserting a gene into a patientâs cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene.
âRetinal gene therapy research shows great potential to, one day, address inherited retinal diseases that currently have no treatment options. It is a very exciting time for patients and families, scientists and clinicians.â â Daniel C. Chung, D.O. Clinical Ophthalmic Lead, Spark Therapeutics.