Gene Modifying Therapy Has Important New Breakthrough

One of the medical research field’s biggest developments recently – within the last 5 years! – is a new form of gene editing, also know as genome editing.

Gene editing refers to a group of technologies that give scientists the ability to change an organism’s DNA.

These technologies allow genetic material to be added, removed, or altered at specific locations.

Several approaches to genome editing have been developed, including clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9, or CRISPR-Cas9.

The CRISPR-Cas9 system has generated a lot of excitement in the scientific community because it is faster, cheaper, more accurate, and more efficient than other existing genome editing methods.

While its potential is unquestionable – ethics and safety concerns have prevented CRISPR from being used to treat human diseases. These ethical concerns chiefly include the ability to edit human embryos.

However, CRISPR/Cas9 is now being studied at the Salk Institute to help treat diabetes, kidney disease, and muscular dystrophy in mice, without cutting DNA.

This method means they avoid unwanted mutations, works epigenetically – or influencing gene activity without changing the DNA sequence.

The Salk Institute made this neat video to explain!

And the studies seem to be working!

The treatment worked in lowering blood glucose levels, targeting acute kidney injury, and treating a form of muscular dystrophy.

And click here to read an interview with Juan Carlos Izpisua Belmonte of Salk’s Gene Expression Laboratory about the new developments!

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