CRISPR gene editing technique holds promise of wiping out genetic diseases

07 May 2015

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A technique called CRISPR (clustered regularly interspaced short palindromic repeats) has brought designer babies a step closer to reality.

The technique was used by geneticists in China who recently modified the DNA of nonviable human embryos and published the results in the journal Protein & Cell.

The first instance of the editing of the genetic material of human embryos was marked by much awe and controversy. 

"While these embryos will not be growing up into genetically modified people," science journalist Carl Zimmer wrote in National Geographic, "I suspect this week will go down as a pivotal moment in the history of medicine."

A number of observers highlighted the potential upsides of this type of DNA modification and one cell researcher told Forbes that CRISPR technology might someday help people "suffering from blood diseases like Beta thalassemia and leukemia, [since they] could have their blood cells' DNA fixed."

According to Paul Knoepfle, an associate professor at UC Davis School of Medicine, CRISPR made him feel like a "kid in a candy store." He could run genetic engineering experiments faster and more cheaply, and could use the technology to better understand the causes of disease.

CRISPR, an advanced gene editing technology can make changes to the genetic code, thus altering the functioning of certain genes.

Researchers at Sun Yat-sen University in China recently admitted to using this technology to try to correct the faulty genetic sequence that led to the fatal blood disorder beta thalassemia.

While many scientists had expressed great concern over the technique, ethics expert Christopher Gyngell argued gene editing was vital, in order to wipe out genetic diseases.

Chinese researchers used human embryos that were left over from IVF and had severe abnormalities, meaning they could never be born.

The rapid speed with which gene editing technologies had developed had taken the scientific community, and many others, by surprise.

In just three years after the development of CRISPR , it has been used to make permanent changes to the genes of yeast, plants, mice, rats, pigs, primates, and now humans.

With CRISPR technology, scientists can precisely target parts of genetic code and unlike other gene-silencing tools, the CRISPR system targeted the genome's source material and permanently turned off genes at the DNA level.

The DNA cut called a double strand break closely mimicked the kinds of mutations that occur naturally, for instance after chronic sun exposure.

However, unlike UV rays that could result in genetic alterations, the CRISPR system brought about a mutation at a precise location in the genome.

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