CRISPR gene technology as told by the lady who invented it

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A TED lecture on CRISPR  Cas9 gene editing technology


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And a bit more on CRISPR

We are living in interesting times.  I mean good interesting.  In the last 100 years, biology, especially biology at the molecular level  has  advanced more than in the previous 5000 years.  

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  • 1 year later...

Headline for a canine cure of muscular dystrophy: CRISPR Gene-Editing Successfully Stops Muscle-Eating Disease In Young Dogs


The gene-editing tool CRISPR has been used to halt muscle dystrophy in a breed of beagle puppies. This brings gene editing one step closer to use in treating human genetic disorders.

In a study published in Science, researchers have reported that using CRISPR, they were able to successfully correct a common genetic disease called Duchenne Muscular Dystrophy (DMD) in young dogs.

DMD results from mutation of a gene that makes a protein called dystrophin.

In puppies, insufficient amounts of dystrophin lead to muscle weakness, clumsy movements and even choking on their tongues – all as early as months after they are born.

The disease also affects humans similarly. People born with the disease are confined to wheelchairs as the muscle degeneration gets progressively worse. In terminal cases, it can even cause vital organs like the lungs and heart failure to stop working.

The four one-month-old beagles that were treated for DMD in the study had their mutant dystrophin gene ‘fixed’ using CRISPR. The researchers cut out a small part of the gene – its mutated section – at a precise location. They then used CRISPR to insert a properly functioning copy of dystrophin wherever it was otherwise mutated and faulty.

Just removing this small section allowed the dogs’ cells to produce enough of the normal protein for healthy muscle function.


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