For millions of people who live with debilitating inherited disease and who don’t want to pass it on to the next generation, scientists have found hope through gene-editing.
Scientist have for the first time corrected a disease-causing mutation in early stage human embryos with gene editing. The gene-editing success holds promise for preventing inherited diseases
US teams at Oregon Health and Science University and the Salk Institute worked with the Institute for Basic Science in South Korea. They focused their work on hypertrophic cardiomyopathy, a disease that affects one in 500 people. It is the most common cause of sudden death in otherwise healthy young athletes.
Here’s the key point:
This work paves the way for eventual cures for thousands of diseases caused by mutations in single genes.
This means the potential end of devastating debilitating diseases like cystic fibrosis, sickle cell disease, muscular dystrophy, or Huntington’s disease.
The embryos, minus the piece of faulty DNA, if allowed to develop fully, would result in babies that would not have the deadly heart disease and would not pass the disease onto their descendants.
What was the scientific breakthrough?
Gene-editing hold risks – it can lead to unintended mutations which can lead to unpredictable consequences. The researchers found a safe method that avoids these dangers.
Science News explains how the researchers avoided mutations and genome instability by editing genes during fertilization instead of after. Previously, scientists fertilized eggs and then added the CRISPR/Cas9 gene editor. Sometimes eggs had already copied DNA, and a mutant gene escaped editing. That led to a patchwork, or mosaic, embryo with edited and unedited cells.
“Gene editing is still in its infancy so even though this preliminary effort was found to be safe and effective, it is crucial that we continue to proceed with the utmost caution, paying the highest attention to ethical considerations,” says Juan Carlos Izpisua Belmonte, a professor in Salk’s Gene Expression Laboratory and a corresponding author of the paper.
The researchers stressed that their results are very preliminary and more research will be needed to ensure no unintended effects.
Altering human heredity to fight genetic diseases is a noble pursuit but one with many ethical implications. Many people, academics and the public alike, have expressed concern about human gene editing, fearing that the same techniques could also be used to enhance intelligence or other physical attributes.
Darren Griffin, a professor of genetics at the University of Kent, stripped the issue down to the fundamentals in an interview with the BBC:
“Perhaps the biggest question, and probably the one that will be debated the most, is whether we should be physically altering the genes of an IVF embryo at all.”
“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.”
What is your take on this? Are we wrong to meddle with genetics or would it be wrong not to use gene-editing techniques if it could prevent unnecessary suffering?