The First FDA Approved Genetic Therapy Now Available, What you Need to Know.

I wrote Gene Therapy Delivered to the Brain, through the Eye right as it appeared that Luxturna was going to be approved by the FDA, back in October of 2017. Funny thing was, I had never heard of Luxturna. I was looking into a study regarding a totally different genetic therapy which was being practiced on mice.

In the mouse study¬† “Eye drops deliver gene therapy for brain disorders,” genetic therapy, effectively delivered genetic material directly to mice brains through their eye balls. Needless to say that creeped me out a bit, so I did a search on genetic therapy, and found Luxturna.

pexels-photo-207585.jpegHere’s the thing, Luxterna is now, the first FDA approved gene therapy. As of March 22nd, 2018, it has just hit the market.¬†Stranger still, its for your eyes. Wild right? Though Luxturna is intended to only change DNA in the eyeball itself, much like the genetic modification of crops. Luxturna uses a virus to deliver genetic material to the hosts DNA, which can be risky to say the least.

I just happened to begin researching this topic again on the same day Luxterna actually became available for sale. No surprise to me, all of the media is positive, and perhaps it should be. This therapy however, is not a time tested treatment. It literally involves sticking a needle deep into the human eye, to deliver a virus carrying genetic material, in order to alter the DNA of the recipient. This is a whole new level of risk.


If the mice studies have anything to tell us, it is that the eyes “at least in mice” are a direct pathway to the brain. This is something that people really need to consider laying down a whooping $425,000 per eye to get this treatment.

Furthermore, there are obvious risks to injecting viruses in to the eyes that can extend beyond the eyes themselves. It could possibly lead to cancers, or other cell dysfunctions. Being that you can’t get much closer to the brain than the eyeballs, these are certainly things to consider.

So what might the consequences of Luxturna be? Well according to Luxturna’s own information here are some of the risks:

  1. Changes in the retina that can lead to vision loss
  2. Eye infections
  3. Permanent decline in vision
  4. Formation of cataracts (which affected 20%)
  5. Swelling
  6. Air bubble in eye
  7. Pain
  8. Pressure
  9. Swelling

In fact, 66% of patients experienced adverse reactions during trial treatments. This means more participants experienced side effects than did not. For a full list of warnings, precautions and risks, visit this site.

pexels-photo-464344.jpegSo before you go looking for rainbows, look for yourself, do some research, and weight your options. Besides being a treatment, so expensive that insurance companies themselves have to set up a payment plan to afford it, it is an overall risky treatment. There are a lot of kinks to work out all without mentioning the moral implications of gene therapy, and DNA alteration in general.

Here’s the deal, it is highly unlikely that anyone reading this will be getting, or know anyone getting the treatment. But, none the less you should remain aware and informed as gene therapy become a normalized medical practice.

All the Best,


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