
Which is an example of gene therapy?
Gene therapy examples include cutting-edge treatments such as:
Luxturna: A gene therapy designed to treat inherited retinal disorders, including Leber congenital amaurosis, which causes blindness due to mutations in the RPE65 gene.
ROCTAVIAN : A gene therapy developed for hemophilia A, which involves the insertion of a functional copy of the clotting factor VIII gene to help restore the ability to form blood clots.
SKYSONA : This gene therapy is used for the treatment of cerebral adrenoleukodystrophy (CALD), a rare and life-threatening neurodegenerative disorder, by delivering a functional copy of the ABCD1 gene.
Zolgensma : A gene therapy designed to treat spinal muscular atrophy (SMA), a genetic disorder that causes progressive muscle weakness, by delivering a functional copy of the SMN1 gene to motor neurons.
These gene therapies represent significant advances in the treatment of genetic disorders by addressing the root causes of these diseases through the delivery of corrective genes.