Gene therapy holds significant promise as an innovative approach to treating and
potentially curing various diseases, particularly genetic disorders. Here are some key
aspects of the promise of gene therapies.
One of the major advantages of gene therapy is its potential to provide long-lasting or
even permanent treatment effects following a single treatment
Unlike traditional drugs that may require ongoing administration, gene therapy aims to
correct the underlying genetic cause of a disease, potentially offering a cure rather than
just managing symptoms with pills and infusions.
Gene therapy shows particular promise for treating single-gene disorders, such as:
• Inherited immune deficiencies
• Certain eye conditions causing blindness
• Blood disorders like hemophilia and beta-thalassemia
• Some cancers, including melanoma and leukemia
For patients with debilitating genetic conditions, gene therapy offers the possibility of
dramatically improving quality of life by addressing the root cause of their disease
This could potentially reduce or eliminate the need for ongoing treatments and disease
management