In the late 1960’s, everyone knew that Dr. Banner was belted by gamma rays, and those turned him into the Hulk. A scientist caught in a lab accident was altered into a huge powerful monster, almost losing his humanity.
The accidental monster was impervious to many of the human weaknesses we take for granted and accept as our destiny.
But should we? From the beginning of human records the death rate from bacterial infection was huge and life expectancy even in the most developed economies was 47 years old. Penicillin and other antibiotics cured that, along with countless other infections. Smallpox erased entire civilizations from history. Vaccinations have pushed that disease to the dustbin of history. No parent today should fear their child’s death or disability from polio, rubella, whooping cough, or smallpox in the United States.
As Dr. Gbola Amusa puts it: “The promise for new genetic medicines, including gene editing, is they can offer durable solutions or even cures for some of the hardest-to-treat diseases in human history.”
Now whole new categories of disease have become treatable — through a range of medicines that use virus ships to infect humans with genetically enhanced cargoes that change the genetic code to eliminate the disease. A real silver bullet.
Luxturna works by delivering a normal copy of the RPE65 gene directly to retinal cells. These retinal cells then produce the normal protein that converts light to an electrical signal in the retina to restore patient’s vision loss. Luxturna uses a naturally occurring adeno-associated virus, which has been modified using recombinant DNA techniques, as a vehicle to deliver the normal human RPE65 gene to the retinal cells to restore vision.
“The approval of Luxturna further opens the door to the potential of gene therapies,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Patients with biallelic RPE65 mutation-associated retinal dystrophy now have a chance for improved vision, where little hope previously existed.”
In other words, a blind person is injected with a new genetic fix that cures blindness.
These advances have been enabled by the power of gene sequencing through incredibly powerful computer chips, a global scientific community that relentlessly pursued these treatments despite incredibly complex biological challenges, and venture capital and public stock investors that have risked hard earned capital in these fantastic biochemistry experients.
These experiments are paying off.
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