Speeches & Floor Statements

Opening Statement: Alexander: Gene Editing Technology, When Used Properly, Has the Potential to Transform Human Health

Posted on November 14, 2017

Eric Lander, a leading geneticist and mathematician who was integral to the Human Genome Project said, “it’s hard to recall a revolution that has swept biology more swiftly than CRISPR”. 

Today, we are looking at this remarkable technology to edit genes that has potential to treat devastating diseases, including those that currently have limited treatments or cures. 

While CRISPR is not the only way to edit the human genome, it is one of the most exciting and talked about ways in the medical research community. 

This is a relatively new technology – it essentially uses molecules that can be targeted to act as scissors to cut and edit genes.

While CRISPR acts as the search function – it goes in and finds the mutated gene, the cas9 is the tool that deletes the disease-causing gene, inserts new genes, or repairs mutated genes.

In a way, it is like cutting and pasting in a computer document.

That may be an oversimplification, but CRISPR technology is less expensive, more precise, and more readily available to scientists all over the world than other gene editing technologies. 

A New York Times story in August reported that CRISPR can be used to do something as frivolous as making yeast glow like Jellyfish to something as serious as making real strides against diseases, such as correcting the gene that causes sickle cell anemia.   

While CRISPR was developed in 1993, its use was perfected for humans in 2013.

Its most widespread use until now has been in agriculture. Disease-resistant wheat and rice has been created using CRISPR, and CRISPR has been used to modify tomatoes and soybeans to improve yields and create healthier soybean oil. 

There is the potential to create crops that can produce higher yields, are able to live through a drought, and have increased nutrition value. Some researchers are even looking at ways to making better tasting crops.

CRISPR’s use in humans is more recent, but the possibility of the diseases it could treat and the lives that could be improved is remarkable.

According to the Centers for Disease Control and Prevention, sickle cell disease occurs in about 1 out of every 365 African-American births. One of our witnesses today will be able to speak to research on how CRISPR can help with this devastating disease. 

Editas Medicine, who is represented by one of our other witnesses today, sees the potential to treat blood disease that today are currently only treatable through blood transfusions and bone marrow transplants. 

Using CRISPR, the genes causing blood disease could be edited and re-administered to treat the disease more safely and effectively.  

For cancer patients, CRISPR could improve the amount of time immune cells are active in fighting tumors. 

The possibilities could go even further – if we could actually could identify the gene mutation that, for example, shows a predisposition to Alzheimer’s, could we edit that gene and prevent the suffering and heartache Alzheimer’s causes? 

While CRISPR and other gene editing technologies could transform human health, it is not hard to see how we can quickly get into societal and ethical issues.

The technology could lead to permanent changes to the human genome, and there is even the possibility of making changes in embryos to create so-called “designer babies.”

In the hands of our adversaries, CRISPR poses national security concerns through the potential to produce new biological weapons. 

In February 2016, former director of national intelligence James Clapper added gene editing to a list of “weapons of mass destruction and proliferation.”

I know that leaders at Oak Ridge National Laboratory and others in the intelligence community are having classified discussions similar to the one we are having today.

Part of our job on this committee is to learn about new technologies, to lead discussions with experts about the implications of these scientific advancements and to ensure that the National Institutes of Health (NIH) and others have the proper authority to oversee and conduct research. 

Part of our job on this committee is to learn about new technologies, to lead discussions with experts about the implications of these scientific advancements and to ensure that the National Institutes of Health (NIH) and others have the proper authority to oversee and conduct research. 

This committee has a long history of working in a bipartisan way to pass legislation that helps advance biomedical research to improve the health of Americans – through the 21st Century Cures Act last year and the reauthorization of the Food and Drug Administration user fees this year.

I am also a member of the Appropriations committee and a strong proponent of increasing funding for basic research – and CRISPR is just one of the amazing discoveries that has come from basic research funded in part by the federal government. 

Senator Murray—who is the Ranking Member on the Appropriations Subcommittee overseeing health research funding—along with Chairman Blunt, have included $2 billion in new funding for three years, in addition to the $4.8 billion we included in Cures, for the National Institutes of Health.

Today’ hearing is truly a hearing, where I intend to do more listening than talking, and I appreciate our panel taking the time to discuss this promising technology today.