Salk Scientists Successfully Edit Genes to Prevent an Inherited Heart Disease

Salk Scientists Successfully Edit Genes to Prevent an Inherited Heart Disease”

It is the first study to use the controversial CRISPR gene-editing technology in a large number of normal human embryos.

This sequence of images shows the development of embryos after co-injection of a gene-correcting enzyme and sperm from a donor with a genetic mutation known to cause hypertrophic cardiomyopathy.

Hypertrophic cardiomyopathy is one of more than 10,000 inheritable diseases caused by an error in a single gene. Today, his research laboratory is investigating the use of CRISPR for modifying disease genes, such as the gene that causes sickle cell disease.

The research marks a major milestone and, while a long way from clinical use, it raises the prospect that gene editing may one day protect babies from a variety of hereditary conditions.

At present, South Korea's ethical guidelines ban experimenting on human embryos in the country.

But while the procedure is considered to be the first of its kind, human trials are not now allowed in the United States.

In the study, sperm from a man who carries a mutation in the MYBPC3 gene was injected into eggs from women with healthy copies of that gene.

A group of scientists in the United States have taken the first steps in tweaking the genes in the human embryo - a subject fraught with fears of an eventual designer baby and a minefield of ethical dilemma. It wasn't a flawless success-the rate of fixed embryos moved from the naturally expected 50% to 74%. The researchers have not attempted to add any new genes or change traits, only to correct a disease-causing version of a gene. In the remaining 27.6% embryos, the cellular cut-repairing mechanism introduced some unwanted insertions or deletions near the cut. That embryo had repaired the mutation in all its cells, but some cells used the mother's DNA for fix while others used the template supplied by the researchers. By successfully testing the technique on human oocytes and sperm, the results of the whole study greatly enlighten researchers when it comes to editing and correcting mutated genes.

The scientists on the call said that while they targeted this particular disease in the experiment, they hope to carry out similar future experiments on other genetic diseases. An example of a legitimate issue regarding genome editing is whether the technology will be safe for clinical use in the near future. Currently, the U.S. Food and Drug Administration is barred from reviewing investigational medical studies involving editing of human embryos - something that would be required in order to proceed with moving this research into practice. Until now only three published Chinese studies have tried CRISPR on human embryos, with mixed results and low efficiency.

The embryos were allowed to grow for five days for analysis.

Embryos' self-healing DNA came as a surprise, because gene editing in other types of cells usually requires an external template, Mitalipov says. In one embryo, the researchers did attempt to insert new DNA, but that embryo experience mosaicism. And groups like the American Society of Human Genetics mostly center their opposition on modifying embryos for implantation into a human.

Earlier this year, an ethics report from the prestigious National Academy of Sciences opened the door to lab research to figure out how to make such changes - but said if germline editing ever is allowed, it should be reserved for serious diseases with no good alternatives and performed under rigorous oversight. However, the goal is to figure out a way to prevent children from being born with genetic diseases - not create designer babies or super soldiers, despite what the guy wearing a tinfoil hat may tell you.

Like this


04 August 2017
Samsung Reveals Dual-Screen high-end Flip Phone, Leader 8
The Leader 8 has been confirmed for a release in China, but if it does expand elsewhere, it will likely only be in parts of Asia. Samsung has even included S Voice instead of Bixby, which is arguably an upgrade over Samsung's unreliable voice assistant.

03 August 2017
Jinder Mahal Reacts to Facing Shinsuke Nakamura at SummerSlam
Owens was not happy but SmackDown Live Commissioner Shane McMahon and General Manager Daniel Bryan understood his frustration. While not official, we can tentatively pencil in a SmackDown tag team title rematch between The New Day and The Usos.

03 August 2017
'It was pretty crazy': Passenger opens aeroplane door and jumps out
Passenger Isaac Rodrigues said they were all told to remain on the plane for over an hour as police dealt with the situation. Relay the message back. "They put one of the stewardesses to block it so no one would fall out of there or leave", he said.

02 August 2017
Sony profits spike with strong segment growth
This significant increase was primarily due to increases in Financial Services and Semiconductors segment sales . The recovery of the image sensor business from last year's quake saw the operation make a profit of $500 million.

01 August 2017
HSBC sees half-year profits rise by 5%
Gulliver said he could be at HSBC as late as December 2018 if an external candidate is appointed by incoming chairman Tucker. HSBC has reported a rise in half-year profit and announced a two billion U.S. dollar (£1.5 billion) share buy-back.

30 July 2017
IRGC Commander Warns of 'Serious Consequences' of US Acts in Persian Gulf
The Americans then left the area, the IRGC added, while the Iranians went on with their patrol. Iran pledged to forge ahead with its missile program Saturday.

28 July 2017
Charlie Gard will be moved to hospice in days
After the judge made his decision, Ms Yates left the court crying and said: "I hope you are happy with yourselves". Great Ormond Street hospital said on Tuesday that would not be possible for practical reasons.

28 July 2017
UK To Ban Petrol & Diesel Cars From 2040
Charging zones for the most polluting vehicles is also an option if these measures don't cut emissions enough. Here are our favourite electric cars now on sale, and we've also named the ones to avoid.

26 July 2017
Donald Trump Blocks Chrissy Teigen On Twitter For This Crazy Reason
Now, it seems Trump has had enough - and it took just five words for Teigen to be blocked by the tweet-happy President. Considering more than 5000 people shared Teigen's response, it's safe to say she wasn't alone in her sentiment.

24 July 2017
Captain Marvel Will Be Fighting Skrulls in the '90s in Her Movie
In fact, fans will remember that back in 2008's Iron Man , Nick Fury told Tony Stark that he wasn't the only superhero around. He did reveal this, however: the enemies Captain Marvel will be battling in her titular movie will be the Skrulls.