Health Care

Heart disease flaw removed from human embryos

Heart disease flaw removed from human embryos”

Genetic diseases are a significant cause of healthcare spending and death globally, and many research groups are using CRISPR as a tool to try to combat them.

A group of scientists in the U.S. 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.

"We need much more basic studies like this to answer the question of whether this is a feasible approach", said Salk staff scientist Jun Wu, who contributed to the study along with Salk researchers Juan Carlos Izpisua Belmonte and Keiichiro Suzuki.

The researchers said more work is needed to ideal the technique and see how widely it could be applied. On the other hand, it highlights ethical dilemmas that we regularly grapple with in reproductive health. It is caused by a mutation in the MYBPC3 gene, but often goes undetected until it is too late.

The researchers used donated sperm from an anonymous OHSU patient with the condition to fertilize eggs donated by 12 healthy women who were recruited in the Portland area through print and online ads.

The news of the successful gene editing technique was first reported last week. But they found that their edits were imprecise. According to a report published Wednesday in the journal Nature, the researchers were able to demonstrate that it is possible to safely and efficiently correct defective genes that cause inherited diseases. Previously, such experiments have been reportedly conducted in China.

Furthermore, to be ethical, any applications or experiments utilizing CRISPR or other gene editing technology can not use any other methods in its process which are themselves intrinsically immoral, Fr.Pacholczyk said.

"Looking at it more closely, it's less useful than you might expect, if it works at all", Greely said.

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This sequence of images shows the development of embryos after injection of a gene-correcting enzyme. Natural DNA-repair mechanisms in the cell follow up by filling in the missing pieces.

Parents could use the scientific advancement to spare their children from a lifelong debilitating disease. It is directed to a specific location in the DNA and performs a cut-and-paste function, not unlike word-processing software. There is also a fear of introducing unintended mutations into germline cells.

The embryos, tested in the laboratory, were allowed to develop for only a few days. Their work was largely funded by private donations and university money. It would also prevent future offspring from inheriting the same condition. They are at a very preliminary stage, but potentially open up the way to major advances in the treatment of genetic diseases. They effectively corrected the problematic sperm gene 72 per cent of the time, and even in those instances where they did not correct it, the problematic gene was still deleted.

Fr. Pacholczyk also stressed the importance of limiting gene editing to therapeutic purposes, with the subject's best interests in mind.

Ethicists told KPBS the researchers have not raised any new problems that were not already known prior to the study's publication. The use of such a technique would inevitably raise the prospect of exerting all kinds of control over human reproduction as well as a host of new ethical questions.

Specialists in the field hailed his accomplishment as the next step in genetic research. But the finding could be good news for those concerned about designer babies, because embryos may reject attempts to add new traits.

"On the other hand, the trajectory is for this technology to be sufficiently robust that it will eventually be used", Kalichman wrote.

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