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Gene Therapy News from UCLA

December 21, 2017

I am certainly no scientist or medical expert, but I am a very determined and hopeful person. I truly believe that we WILL see a cure for Friedreich's Ataxia in my lifetime, and I think it will be from gene therapy. Therefore, any news I see related to gene therapy advancements (through FA research, or that of other diseases) I get excited.

 

However, one of my biggest fears is the time clock I am up against. Every day, I am progressing little more. Every day, my central nervous system is a little more damaged as FA takes over my body. How reversible is that damage? Will I every be "normal" again? Unfortunately, there is no pause button for my progression while the researchers work on a cure for me. Therefore, my fear is that the damage is irreversible. What if they cure FA, but it just means I won't progress further, and not get better? See my point? 

 

The latest news from UCLA addresses that fear exactly. 

UCLA researchers, after developing a mouse model of Friedreich's ataxia that shows symptoms similar to patients, have found that many early symptoms of the disease are completely reversible when the genetic defect linked to the ataxia is reversed. The new findings, which still need to be replicated in humans, appear in the journal eLife.

 

"Remarkably, most of the dysfunction we were seeing in the mice was reversible even after the mice showed substantial neurologic dysfunction," said Dr. Daniel Geschwind, the Gordon and Virginia MacDonald Distinguished Chair in Human Genetics, a UCLA professor of Neurology and Psychiatry, and senior author of the new work. "We were very surprised by the extent to which the mice improved since we had assumed that this degree of behavioral dysfunction would be due to cell loss"

 

THIS IS INCREDIBLE!! Now, they just need to hurry up and start human trials so we can all move on! To read the full write up, click here

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