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First US Human Embryo Gene Editing Experiment Successfully "Corrects" a Heart Condition

First US Human Embryo Gene Editing Experiment Successfully

For the first time in the United States, researchers have used gene editing to fix a mutation in human embryos.

According to an article published by American Health Line earlier this week, a team of biologists in OR have conducted the first known experiment in the United States using the gene-editing technique CRISPR to genetically modify viable human embryos.

For the first time, researchers in the US have safely repaired a disease-causing gene in human embryos, targeting a heart defect best known for killing young athletes - a big step toward one day preventing a list of inherited diseases.

With advances in stem cell technologies and gene editing, researchers are finally starting to address disease-causing mutations that impact potentially millions of people.

Today, people who carry certain genetic diseases can opt for IVF and have their embryos screened for harmful mutations but there are many ethical questions about the potential for creating "designer" babies that could be "edited" to have a specific intelligence, height, facial appearance or hair colour. "Consent is particularly important when dealing with very vulnerable research subjects, and human embryos are among the most vulnerable of God's creatures".

The experiments were privately funded; US tax dollars aren't allowed for embryo research.

In 2015, scientists in China conducted the first known attempt to use CRISPR on human embryos.

CRISPR-Cas9 works as a pair of genetic scissors created to cut the DNA near the position of the mutation and correct the hypertrophic cardiomyopathy mutation carried in the DNA of the sperm, it said.

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But in an interview with KPBS, a Salk scientist involved in the study said more scientific work and ethical consideration is needed before using this approach in the creation of any future human children.

The experiment proved successful in preventing the spread of a disease that usually has a 50 per cent chance of being passed on in 42 of 58 embryos. The defective gene was cut out using CRISPR around the time the sperm was injected into the eggs. Then, the cut is spontaneously repaired by the cell with different mechanisms: one repairs the DNA without leaving any trace, while the other introduces some unwanted insertions or deletions of a few base pairs near the cutting site.

The researchers found no changes to the embryos' genome outside the targeted mutation. Scientists are also concerned about the unregulated genetic engineering may lead to a new form of eugenics- improving a baby by controlled breeding to increase the occurrence of desirable heritable characteristics.

"It's an incremental step that moves us a little closer [to editing human embryos], but this [technology] isn't right around the corner", Stanford bioethicist Hank Greely told Gizmodo.

As the authors themselves noted, many couples use pre-implantation genetic diagnosis to screen embryos at fertility clinics, allowing only healthy ones to be implanted. Last year, the U.S. Congress ruled against using CRISPR in clinical trials to edit embryos.

"What it does is, it searches out the one error in the 3 billion letters of the DNA code and it chops it out, and the DNA corrects itself", CBS News medical contributor Dr. David Agus explained on "CBS This Morning".

Tweaking the genes of human embryos that could theoretically be brought to term and grown to adulthood is a feat that sounded like science fiction - until this week.

"Although it implies a future in which we could choose to have children without particular genetic diseases, or even design for particular traits, that is still likely a very long time from now", he wrote in an email to KPBS.

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