How Controversial Gene Editing Could Lead To Groundbreaking Cures

Thanks to the controversial new technology known as CRISPR, scientists are beginning to make headway in understanding and potentially curing some of the world’s most intractable diseases.

Sickle-cell anemia, HIV, schizophrenia and autism — essentially, anything involving bad DNA is now fair game. The latest example, from a study published earlier this month in the journal Molecular Therapy, focuses on Facioscapulohumeral muscular dystrophy, or FSHD, which is one of the most common forms of muscular dystrophy. The genetic disease causes the muscle fibers in the face, shoulders and upper arms to weaken over time — and there is no known cure.

Enter CRISPR. This new gene-editing technique allows researchers to easily change, delete or replace genes in any plant or animal, including people. Picture the precision and ease of the find-and-replace function on a word document — that’s how easy it now is to change the human genome. As an article in the MIT Technology Review put it last year, “This means they can rewrite the human genome at will.” Or, as one bioethicist told The Huffington Post last week, comparing what CRISPR can do to earlier attempts at genetic manipulation, “We used to have a butter knife, now we’ve got a scalpel.

Biomedical researchers all over the world are now wondering how the technology might change their approach to all sorts of diseases. About a year ago, a team of FSHD researchers, led by Peter Jones at the University of Massachusetts Medical School, decided to give CRISPR a try. They already had a pretty good idea which of the thousands of genes in the human genome caused the disease, but until CRISPR came along therapeutic avenues were limited.

The acronym CRISPR, which stands for (take a deep breath) “clusters of regularly interspaced short palindromic repeats,” refers to —> Read More