In another win for science, researchers at Oregon Health and Science University (OHSU) successfully performed gene editing on human embryos, removing the gene that causes hypertrophic cardiomyopathy—a disease that affects roughly one in 500 people.
If born, babies of these embryos would be free of the heart condition and also not pass on the mutation to their children.
Although researchers do not plan to transfer embryos from the study to women, this research is the first step to using similar genetic editing techniques in reproductive medicine, such as in vitro fertilization (IVF) treatments.
Does this new advancement mean “designer babies?”
Not at all.
This cutting-edge technology means doctors and patients will have more tools to use when fighting and preventing disease. The technology is nowhere close to being able to edit the genes that determine physical characteristics, intelligence, or athletic abilities, for example.
SGF fertility expert Dr. Stephen Greenhouse offers his perspective on the recent OHSU study and explains what it means for our patients.
Gene Editing and IVF
Think of gene editing as high-tech scissors—if there’s a gene that causes a disease, a doctor would be able to remove the abnormal gene from DNA and then replace those genes with a normal one to prevent a disease from occurring.
Right now, performing this procedure on an egg or sperm is practically impossible to do without also causing damage. Instead, gene editing is carried out on very early-stage embryos, shortly after fertilization.
The benefit of genetic editing in combination with traditional IVF methods is that more healthy embryos are created.
For instance, suppose a couple comes to SGF for fertility treatments, and both partners are carriers of the cystic fibrosis gene. There is a 25 percent chance that their baby will have cystic fibrosis.
In the IVF process, our doctors genetically screen the embryos prior to transfer to identify those with two abnormal genes, which would indicate cystic fibrosis in the embryo.
Currently with IVF and pre-implantation genetic diagnosis (PGD), we can identify those embryos that would be unaffected with cystic fibrosis. On average, one in four embryos would not be suitable for transfer.
With gene editing and IVF, the parents and their doctor wouldn’t have to choose which embryos to transfer as doctors could remove the abnormal genes in the affected embryos, thereby increasing the number of healthy embryos ready for transfer.
Fighting Genetic Disease at the Source
The OHSU study specifically targeted the gene for hypertrophic cardiomyopathy. But scientists could use the same gene editing technology to target other genetic diseases and disorders such as:
People with these and other genetic diseases would be good candidates for gene editing treatment when it becomes available. However, gene editing would only be an option for people with a known genetic abnormality for which scientists know the specific gene that causes the disorder.
Even still, human gene editing isn’t the answer for every illness.
For example, hypothyroidism is easily treated with medication, so a difficult and invasive procedure to cure it would not be necessary. On the other hand, if a devastating illness such as Tay-Sachs disease is detected, genetic editing may be worthwhile.
Research Today, Medical Treatment…Tomorrow?
As of today, genetic-editing technology is in the early stages of development with procedures happening only in research settings, not in clinical ones.
In fact, the U.S. Food and Drug Administration (FDA) requires special permission for genetically edited embryos to be transferred into a womb.
Right now, the FDA isn’t funding this research.
Though there is not a ban on human gene editing in the U.S., the lack of funding means progress will be slow.
Researchers must present their studies to institutional review boards (IRBs) to ensure that their methods are safe and ethical.
Several more years of research are needed before genetic editing technology is ready for use with patients. Even when the technology becomes available, the question of cost remains.
Will insurance cover human gene editing? Will the procedure only be available to higher income families? Our goal at SGF is to make treatment available to all and to remove the barriers that stand in the way of creating a family.
Research like what’s taking place at OHSU and other facilities will open up new avenues of treatment for our patients, leading to refinements of the treatments we currently offer at Shady Grove Fertility.
It’s exciting that in the future we may be able to transfer a higher percentage of embryos and increase the chance of conception for our patients.
To learn more about gene editing and IVF or to schedule an appointment with one of our 40+ reproductive endocrinologists, please call our New Patient Center at 1-877-971-7755 or click here to complete this brief online form.