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Genetic Therapies Promise Cures To Inherited Disease

University of Utah
University of Utah

The University of Utah has been a leader in the field of genetics research for decades. Ethical issues are on the rise as the technology now allows genome editing of human embryos.

University of Utah Pediatric Professor and Associate Vice President of Research Jeffery Botkin says the years of genetics work is now moving into a new phase that is treating and curing some diseases through gene technology.

“The University of Utah has really been the leader in the genetics field for decades. And due to the wonderful collaboration with our community that tends to have large families and tends to track those families over time. And that’s enormously helpful for understanding genetics."

Genetic mutations can lead to diseases like sickle cell anemia and cystic fibrosis. They’re caused by single gene defects passed from parent to child. Botkin says it’s estimated that 40 percent of children in hospitals are being treated for genetic conditions and he says they’re making headway in tackling these genetic illnesses.

“There’s a really sophisticated way to intervene with genes and to actually cut out abnormal genes and potentially add in new genetic material if you know what you’re targeting. It’s turning out to be an exciting time from that respect. But from my perspective and a lot of people’s perspective, some challenging ethical issues as we decide how to use this powerful technology in the right way.”

He says the National Institutes of Health sets aside a significant amount of money for ethics related research. Crispr technology treats genetic mutations in the lab by altering the cells and reintroducing them into the patient. He says there are clear ethical issues that come with altering cells in humans and human embryos.

“Some of the key issues just relate to safety. Are you getting those genes into the right place and are they working the way you want them to. If you’re making a mistake, part of the challenge is, it’s pretty tough to get those genes out of there again. Once you have a gene inserted in your body, it’s pretty tough to get that out. So, those are some of the ethical challenges mostly around safety and research ethics that are part of that.”

Botkin says Medicare recently approved a genetic therapy called Car T Cell which is a cancer treatment. He says it is potentially a single treatment cure for certain types of cancer.

“Very exciting. It looks like a number of people have been cured with this treatment. It’s been now approved. A significant issue is the cost. And the cost is between 375,000-$475,000 per treatment.”

Botkin says another significant breakthrough is a genetic therapy for spinal muscular atrophy estimated to cost $2.1 million for one treatment.

“And the more severe form will lead to a child’s death in the first couple years of life through progressive weakness. Now, it looks like there is a genetic therapy for this that may well be extremely helpful if not curative. The fact that Medicare has approved this Car T Cell treatment for cancer is encouraging that that will be available for folks who meet the criteria for this.”

Botkin says as more therapies come on-line, the issue of accessibility to expensive genetic treatments will be a public conversation and he’s not sure how it will turn out.

He says the University of Utah has a sophisticated and highly visited website called the Genetic Science Learning Center. He says it’s a great way to understand genetics and how the gene editing technology and treatments are progressing.  https://learn.genetics.utah.edu/content/basics/

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