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Assistant professor of biochemistry Jill Wildonger, back, and research specialist Dena Johnson-Schlitz, left, work with fruit flies (drosophila) in a laboratory at the DeLuca Biochemical Sciences Building at UW-Madison in this image from May 3, 2016. Wildonger is using the CRISPR-Cas9 gene editing technique with the flies. 

M.P. KING -- State Journal

Don’t expect designer babies any time soon, but a major new ethics report with key input from UW-Madison faculty leaves open the possibility of one day altering human heredity to fight genetic diseases using new tools that precisely edit genes inside living cells.

The report Tuesday from the National Academy of Sciences and National Academy of Medicine was compiled by a 22-member committee with two members from UW-Madison: R. Alta Charo, a professor of law and bioethics who co-chaired the NAS panel, and Dietram Scheufele, a professor of life sciences communication.

What’s called genome editing already is transforming biological research and is being used to develop treatments for patients struggling with a range of diseases.

The science is nowhere near ready for what would be a huge next step — altering sperm, eggs or embryos to prevent babies from inheriting a disease that runs in the family, the report said.

But if scientists learn how to safely pass alterations of the genetic code to future generations, the panel said “germline” editing could be attempted under strict criteria, including only targeting serious diseases with no reasonable alternative under rigorous oversight.

“Caution is absolutely needed, but being cautious does not mean prohibition,” Charo said.

“This committee is not saying we will or should do germline — heritable — editing,” she added. “What we are saying is that we can identify a set of strict conditions under which it would be permissible to do it. But we are far, far away from being ready to try.”

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Alta Charo (copy)

 Alta Charo 

Genome editing should not go beyond healing the sick to enhance traits such as physical strength, what’s commonly called “designer babies,” the panel stressed.

But the public should get involved in these debates now, to say what might one day be acceptable.

“We want to bring in people even if they know little about the topic and get them to the point where they engage in meaningful debate,” Scheufele said in a statement. “Public debate will blur the lines between this issue and others but we want to move forward in a responsible fashion with the best available science and a meaningful understanding of the risks and benefits.”

The long-awaited report offers advice; the prestigious academies do not set policy. But it is considered a step toward creating international norms for responsible development of this powerful technology. The U.S. National Academies and its counterparts in Britain and China have been holding international meetings with the hope of doing just that.

“Genome editing is a new tool for gene therapy and it has tremendous promise,” Charo said. But, she added, it has to be pursued in a way that promotes well-being and is responsible, respectful and fair.

Genome editing is essentially a biological version of cut-and-paste software, allowing scientists to turn genes on or off, repair or modify them inside living cells. There are a few older methods but one with the wonky name CRISPR-Cas9 is so much faster, cheaper and simpler to use that it has spurred an explosion of research.

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Gene editing

Under development are ways to treat a range of diseases from sickle cell and hemophilia to cancer. In lab experiments using human cells or animals engineered with humanlike disorders, scientists are unraveling how gene defects fuel disease — and are even trying to grow transplantable human organs inside pigs.

That kind of research is very promising, is adequately regulated today and should continue at full speed, the National Academies panel concluded.

When it comes to the more sci fi-sounding uses, it’s possible scientists will learn how to perform germline editing in five to 10 years, said panel co-chair Richard Hynes of the Massachusetts Institute of Technology. Safety is one reason for caution, he said, as scientists will have to learn whether editing one gene has unwanted effects.

Some critics argue that families plagued by inherited diseases already have other alternatives — adoption, use of donated eggs, or undergoing in vitro fertilization and discarding any resulting embryos that inherit the bad gene. But Charo noted that sometimes parents carry two copies of a lethal gene, guaranteeing any children inherit it. Others oppose discarding of embryos for religious reasons.

For some families, “you can see there would be strong arguments for doing it” if the other criteria are met, said Robin Lovell-Badge of Britain’s Francis Crick Institute.

Some countries prohibit any germline editing research. Others, such as Britain, allow laboratory research to understand human development.

In the U.S., scientists can perform laboratory embryo research only with private funding. Any attempt at pregnancy would require permission from the Food and Drug Administration, which is currently prohibited from using federal funds to review any such request.

“The bottom line is there is no planetary government with enforcement power,” Charo noted Tuesday.

State Journal reporter David Wahlberg contributed to this report.

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David Wahlberg is the health and medicine reporter for the Wisconsin State Journal.