(IJCH) CRISPR - Successfully treating canine muscular distrophy. One step closer towards human treatments?
IJCH - Inside JaiChai's Head (Meaning: My Warped, Personal Opinions and Musings)
From the Author:
Salutations.
I am JaiChai.
And if I haven't had the pleasure of meeting you before, I'm delighted to make your acquaintance now.
I invite you to interact with everyone, learn, and have as much fun as possible!
For my returning online friends, "It's always great to see you again!"
CRISPR
“CRISPR” (pronounced “crisper”) stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology.
I have written about CRISPR before in these articles:
Article Link:
https://steemit.com/science/@jaichai/ijch-crispr-humanity-s-salvation-or-its-ultimate-pandora-s-box
Article Link:
CRISPR - Successfully treating canine muscular distrophy. One step closer towards human treatments?
A rare form of muscular dystrophy called "Duchenne muscular dystrophy" is a rare, debilitating disease that predominantly affects males (usually during childhood through early adulthood).
Approximately 300,000 people around the world are victims of this disease annually.
Those diagnosed with Duchenne muscular dystrophy do not live very long - usually dying in their 20's from the complications of heart failure.
But the successful treatment of muscular distrophy in Beagles provides hope for human treatments in the near future.
See the following articles:
The powerful gene editing technology CRISPR is one small step closer to treating a human disease.
In a new paper published in Science, researchers led by Eric Olson, professor and chair of molecular biology at UT Southwestern Medical Center, reported that he and his team successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles bred with the disease-causing gene.
It’s the first use of CRISPR to treat muscular dystrophy in a large animal. (Previous studies had tested the technology on rodents.) In varying degrees, the genetic therapy halted the muscle degradation associated with the disease.
CRISPR, the genome-editing tool, introduced a mutation in a dog gene that, in effect, overrode a mutation responsible for a disease that mimics Duchenne muscular dystrophy (DMD).
As a result, muscle cells in the dogs began to produce the dystrophin protein in many tissues, including the diaphragm and the heart.
The results, though preliminary, bring scientists one step closer to making such treatments a reality for humans, says study coauthor Eric Olson, a molecular biologist at the University of Texas Southwestern Medical Center in Dallas.
By JaiChai
Mighty Kind of You for stopping by.
Truly hope to see you again!
About the Author
Believing that school was too boring, he dropped out of High School early; only to earn an AA, BS and MBA in less than 4 years much later in life – while working full-time as a Navy/Marine Corps Medic.
In spite of a fear of heights and deep water, he performed high altitude, free-fall parachute jumps and hazardous diving ops in deep, open ocean water.
After 24 years of active duty, he retired in Asia.
Since then, he's been a full-time, single papa and actively pursuing his varied passions (Writing, Disruptive Technology, Computer Science and Cryptocurrency - plus more hobbies too boring or bizarre for most folk).
He lives on an island paradise with his teenage daughter, longtime girlfriend and three dogs.
