CRISPR surgery for inherited retinal diseases: landmarks in the 21st century

Cell & Gene Therapy Insights 2019; 5(11), 1451-1456.


Published: 18 November 2019
Expert Insight
Stephen H Tsang, Alexander Chai

Gene therapy was first conceptualized in 1972 as clarification on viral DNA-altering mechanisms was done. Since then, the field of gene therapy has transformed from a biological fantasy into a valid clinical treatment in humans, in part due to significant innovations in the field of molecular genetics. The development of gene therapy technology and the ensuing research has laid a strong foundation for the advancement of gene therapy, which has the potential to correct dominantly inherited disorders that were previously incurable. In November 2018, a drug named Luxturna became the first in vivo CRISPR/Cas9 genome surgery treatment to be FDA-approved for use in clinical trials, which are set to take place in patients with Leber congenital amaurosis 10 in the fall of 2019 [1]. However, there remain a number of scientific and practical barriers to resolve before genomic medicine can become a widespread treatment.