First embryo gene-repair holds promise for inherited disease: Research

First human embryos edited in the USA. Here's why it's problematic.

Humanity has an ethical question it's going to have to think about now with today's announcement that scientists in OR have successfully edited genes in human embryos to fix a disease-causing mutation that often kills young athletes.

In a world first, United States researchers announced on Wednesday in the science journal Nature they had used the controversial gene editing technique, CRISPR-Cas9, to correct a mutation for a heart condition in embryos. This genetic disease, which occurs in one out of 500 people, can cause sudden death and a host of other cardiac problems such as heart failure and arrhythmias. Furthermore, by cutting out a gene that caused a heart defect, the disease (called hypertrophic cardiomyopathy) would theoretically also be prevented from being passed down from generation to generation.

In a groundbreaking experiment, an worldwide team of scientists on Wednesday officially reported the successful elimination of a genetic disease from human embryos.

An worldwide group of 11 organizations Wednesday issued a political statement against the changes to the genome ends with implantation and pregnancy, at the same time supporting research, financed from the state budget, the potential clinical applications.

The research on human embryos is strictly regulated and there was no question of implementing those of the study in a woman's uterus to initiate a pregnancy.

CRISPR-Cas9 cut the DNA at the correct position in all tested embryos (100%) and 42 out of the 58 embryos (72.4%) did not carry the hypertrophic cardiomyopathy mutation.

The scope of the research to date is far more limited: In earlier experiments, Chinese scientists were able to make changes to the genetic code of human embryos, but there was a high incidence of off-target effects.

Prior to what has been called a "milestone" in the US, the only country to edit embryos successfully was China. The researchers have not attempted to add any new genes or change traits, only to correct a disease-causing version of a gene.




The team said: "Mosaicism in gene-targeted human embryos is unacceptable in clinical applications". The Salk scientists contributed by developing the gene editing strategy, initially testing it in stem cells derived from the patient's skin cells.

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.

Unlike past experiments that attempted CRISPR days after embryos had developed, this one did it at the time of fertilization. Think of it as a biological cut-and-paste program.

In other cells in the body, the editing process is carried out by genes that copy a DNA template introduced by scientists. 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.

However, the new research, reported in the journal Nature, goes much further by modifying the nuclear DNA at the heart of the cell that influences personal characteristics such as height, facial appearance, eye colour and intelligence.

Genetics and ethics experts not involved in the work say it's a critical first step - but just one step - toward eventually testing the process in pregnancy, something now prohibited by USA policy. The work is also consistent with recommendations issued in February 2017 by the National Academy of Sciences and the National Academy of Medicine joint panel on human genome editing.

Meanwhile, critics worry about attempts at "designer babies" instead of just preventing disease.

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