Disease gene 'edited' in human embryos

Shoukhrat Mitalipov is the first U.S.-based scientist known to have edited the DNA of human embryos.		
	OHSU  Kristyna Wentz-Graff

Shoukhrat Mitalipov is the first U.S.-based scientist known to have edited the DNA of human embryos. OHSU Kristyna Wentz-Graff

The trial was conducted by an Oregon Health and Science University researcher, Shoukhrat Mitalipov. But that may require several cycles of IVF, which is expensive and carries with it side effects and complications, before enough genetically healthy embryos are created.

The Salk/OHSU team also found that its gene correction did not cause any detectable mutations in other parts of the genome - a major concern for gene editing. Induced double-strand breaks (DSBs) at the mutant paternal allele were predominantly repaired using the homologous wild-type maternal gene instead of a synthetic DNA template. It has no cure and can be caused by inheriting just one copy of the bad gene.

The technique could be relevant to a huge number of couples in thousands of inherited diseases caused by heterozygous mutations, where one partner has a normal allele.

The first published work involving human embryos, reported in 2015, was done in China and targeted a gene that leads to the blood disorder beta thalassemia.

Currently, the National Institutes of Health does not fund research into altering the human germline for clinical purposes and the FDA is prohibited from considering any clinical trial relating to germline genetic modification. MYBPC3 mutations account for approximately 40% of all genetic defects causing HCM and are also responsible for a large fraction of other inherited cardiomyopathies, including dilated cardiomyopathy and left ventricular non-compaction6.

An account of the research, complete with photos of the multiplying cells, was published in the prestigious British journal "Nature". Mitalipov's team found no such off-target effects, a sign that CRISPR editing, at least in this study, was relatively safe. The mutation associated with hypertrophic cardiomyopathy is in the MYBPC3 gene. It does not manifest until adulthood and affects one in 500 adults, the paper said.

The gene mutation in question causes a hereditary disease called hypertrophic cardiomyopathy which causes the wall of the heart muscle to become thickened.

The human embryos used in the research were created using eggs collected from healthy women and sperm from a man carrying a DNA error.

But the new study, published in Nature, suggests that CRISPR might work as an aid to fertility clinic screening for unsafe inherited diseases, relying on a previously unknown ability of human embryos to swiftly fix their genes.

"It was easy", Mitalipov said. The hope was the embryos would then fix the cuts with healthy versions.

The embryo used the healthy gene from the female donor as a blueprint to correct the mutant gene. This is why scientists have not been allowed to develop over a few days. There is still no indication as to how they might fare if actually implanted in a human womb.

Lanner is also editing genes in human embryos, as a way of learning more about developmental biology. They were not intended for implantation either. The study that was conducted in USA comes just months after a national scientific committee recommended new guidelines for modifying embryos, easing blanket prescriptions but urging the technique be used only for dire medical problems, the report added.

Aside from helping the United States catch up to China in the genetic arms race, the study breaks some important new ground. "I think it will be up to them to decide where to draw the line".

The term stems from the idea that parents will be able to select the genes they want and don't want in their babies, stoking fears of a Gattaca-like scenario where society is divided into the genetically enhanced and the genetically poor.

"It is a very important study", said Dr. Dusanka Babovic-Vuksanovic, genomics chair at Mayo Clinic in Rochester, Minnesota. But he notes that in Sweden, it would be illegal for him to create embryos exclusively for the sake of research.

"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. Her boy is a carrier, but her girl, Sofia, has the disease. Homozygous MYBPC3mutations in adults are extremely rare owing to the severity of the clinical symptoms and early onset of the disease.

Mitalipov hopes that their technique could one day be used to treat a wide-range of genetic diseases and save the lives of millions of people.

"We need to see if this can be replicated and evaluate the safety", said Wu.

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