Health Care

Designer Baby On Way? US Scientists Modify Genes Of Human Embryo

Designer Baby On Way? US Scientists Modify Genes Of Human Embryoā€¯

The procedure was conducted on embryos created in the lab for the objective of testing the CRISPR-Cas9 treatment.

USA scientists have succeeded in altering the genes of a human embryo to correct a disease-causing mutation, making it possible to prevent the defect from being passed on to future generations.

"I am quite sure that there are tools that we could use to improve. this fix so that we could achieve say 90 to maybe 100-percent efficiency, then I would say we would be ready to move to clinical trials", said Amato's colleague Shoukhrat Mitalipov.

Congress has enacted barriers to clinical trials for such genetic trials, and the National Institutes of Health has banned funding.

CRISPR stands for clustered regularly interspaced short palindromic repeats.

Scientists invented a tool CRISPR-Cas9 works as a type of molecular scissors that can be selectively cut away unwanted portions of the genome and replace them with new DNA.

Among key questions: Would the technique work if mom, not dad, harbored the mutation? Revised code can then be inserted into the snipped DNA.

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Led by Shoukhrat Mitalipov of Oregon Health & Science University, the team injected eggs from healthy donors with sperm from a donor with hypertrophic cardiomyopathy, a common condition characterized by irregular heartbeat and heart failure.

The process was tested on 18 lab-created embryos using sperm from the male donor and eggs donated by 12 healthy young women, the study said. In the USA, taxpayer funds can not pay for research that destroys human embryos.

If a feat similar to that seen in this experiment could be achieved in an afflicted embryo that was allowed to develop into a person, it would prevent the condition in this individual.

The researchers found that 72.4 percent of the embryos, not 50 percent as would have been expected, were free of the errant gene.

"There are still a lot of safety issues, a lot of legal and ethical issues", even in medical uses, said Michael Werner, executive director of the Alliance for Regenerative Medicine in Washington, D.C. The group promotes the use of gene therapy to treat diseases, but has drawn a line at treating embryos.

However, the 16 embryos that did not become free of the mutation experienced unintended genetic deletions or insertions. The tool used is the technique CRISPR-Cas9, a major discovery unveiled in 2012.

"You could certainly help families who have been blighted by a awful genetic disease", said Robin Lovell-Badge, a professor of genetics and embryology at the Francis Crick Institute in London, who was not involved in the study.

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"This is not only a different technique for embryo editing, but the most encouraging fixing of an embryo with CRISPR to date", Eric Topol, a geneticist at Scripps Research Institute who was not affiliated with the study, told Gizmodo.

"This is not a straightforward question... equally, the debate on how morally acceptable it is not to act when we have the technology to prevent these life-threatening diseases must also come into play".

But there is no shortage of concerns regarding genome editing.

The regulation of future CRISPR research in the United States remains unclear as well.

However, the team also noted that genome editing approaches must be "further optimized" before moving to clinical trials. That's encouraging for one potential use of CRISPR in the future as a way to correct inherited genetic disease, says Mitalipov, since the embryo seems to have a built-in, reliable way of repairing the injury caused by splicing out an abnormal gene.

"There is a long road ahead, particularly if you want to do this study in a regulated way", said Mitalipov.

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