Medicine

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.



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