After a long wait, researchers have finally been granted funding for further research and development of the World’s first treatment for a fatal neurological disease called Friedreich’s Ataxia. Friedreich’s Ataxia is a neurodegenerative disease that occurs from a recessive inherited gene that progressively damages the nervous systems and causes movement problems due to impaired muscle coordination or “ataxia.”
Geneticists have brought Dr.Marek Napierala onto the team that is planning on developing the first treatment (and hopefully cure) for FA among other neurological diseases such as Parkinson’s Disease and Huntington’s Disease.
CRISPR has shown promise in the research on mice that found CRISPR could reverse Huntington’s Disease.
From culture to cure
CRISPR is short for Clustered Regularity Interspaced Short Palindromic Repeat which is a powerfully simple tool used for editing genomes. It consists of 2 molecules that introduce a mutation into the DNA.
CRISPR can be attributed to Rodolphe Barrangou and his team of researchers that were studying active yogurt bacteria cultures. His team of researchers found that CRISPR fought off the bacteria commonly found in yogurt cultures.
CRISPR controversy
The controversy surrounding CRISPR is caused by the practice of genetically modifying and experimentation on embryos. CRISPR research on embryos has shown that it could be theoretically possible to edit the genome of an embryo of a child who may carry faulty genes and repair the embryos DNA code.
However, Congress has largely banned funding embryo testing due to mosaicism and because of the fact, the US National Institute of Health does not support embryo research at this time.
Researching Cas9: hoping for a cure but being realistic
New research is leaning towards the possibility that geneticists may be able to customize variants of Cas9.
Whereas, not long ago, genetic research involved the use of hazardous chemicals and radiation to mutate genes. This was detrimental not only to research but also to any subject they tried to do case studies on.
Despite all the positive attention that CRISPR is getting, it is important to be realistic. Geneticists want people to know that CRISPR is not 100 percent effective all the time.
However, it is a viable option that will not break the bank for people with FA among other degenerative diseases. If there is a chance, speaking for myself among others who have a rare and fatal neurodegenerative disease, it is worth the effort and time.
