facebook linkedin twitter

Developer of Gene Editing Tool Discusses Ethics of Emerging Treatments

October 20, 2021 by Alexa Hornbeck
Developer of Gene Editing Tool Discusses Ethics of Emerging Treatments
Jennifer Doudna

It was only nine years ago that researchers discovered a method for editing human genes using a specialized technology called the CRISPR-Cas9 tool.  

CRISPR-Cas9 enables geneticists and medical researchers to edit parts of the genome by removing, adding or altering sections of the DNA sequence. 

Ethicists, however, question whether such manipulation of the building blocks of life could cause future generations to suffer from unexpected mutations of an edited gene and a host of possible physical maladies as a result.

“There are absolutely ethical dilemmas in gene editing, some that are shared across medicine, and others that are novel because we haven’t fully grappled with the concept of changing the human genome until now,” said Jennifer Doudna, co-developer of the method for genome editing, in an email to The Well News. 

In 2012 Doudna and Emmanuelle Charpentier, director at the Max Planck Institute for Infection Biology in Berlin, collaborated on adapting an earlier version of the CRISPR tool to cut specific sites of isolated DNA.

Afterwards “one of the first thoughts was how we might apply CRISPR to treat diseases like sickle cell disease. We had known the genetic basis [of the disease] for a long time, but never had the tools to address it,” Doudna said. “But we were quickly faced with a series of questions: How do we target specific cells in the body and make sure there are no unintended effects? How do we deliver CRISPR inside of those cells? How do we ensure that there is no immune response to the therapy?” 

Doudna said that one of the appealing features of genome editing is that many of the steps to creating a treatment are the same whether you’re treating sickle cell disease, congenital blindness or any other genetic disease. 

“The toolkit is the same, only the target in the genome changes. Like how the assembly line revolutionized auto manufacturing, the same type of thinking can be applied to developing CRISPR cures. Does the regulatory world think along those lines? No, not yet. Have we perfected all of the components for that assembly line? No, not yet,” Doudna said. 

However before those questions could be fully answered, the technology had already begun to be used by scientists. 

In 2013 researchers from the Broad Institute, a collaboration between Harvard University and MIT, started the process of using the CRISR-Cas9 tool in plants, animals and human cells.

It wasn’t until 2015 when the first report of genes edited in human embryos led by Junjiu Huang, a gene-function researcher at Sun Yat-sen University in Guangzhou, ignited a global debate about the ethics of using gene editing technologies. 

In 2017, an advisory group formed by the National Academy of Sciences and the National Academy of Medicine endorsed gene editing but under the condition that it be used to treat only serious diseases and disability and only when no reasonable alternative exists. 

In 2018, a Chinese biophysicist He Jiankui announced that twin girls, named Lulu and Nana, were born from embryos that he and his colleagues had edited to make them less susceptible to HIV.

The following year Jiankui and his colleagues were sentenced to three years in prison by the Shenzhen Nanshan District People’s Court and barred from working in reproductive technology based on the ethical issue of prematurely introducing a mutation that could potentially cause harmful effects and produce little benefit, as there was no significant risk of HIV. 

Jiankui’s case prompted a group of more than 60 individual scientists, bioethicists, and biotechnology executives to call for a global moratorium on human clinical germline experimentation in a letter to the U.S. Department of Health and Human Services.

Beyond the therapeutic applications of the CRISPR-Cas9 tool, many scientists worry about the possibility of these treatments being used to enhance human traits, such as strength, or cognitive capacity, which can be done by targeting cells in adults or embryos.

“The ethics become less complicated when you focus on gene editing therapies in individuals, which is our focus at the Innovative Genomics Institute, as opposed to heritable gene editing,” said Doudna. 

Doudna also raised the ethical question of who would have access and be able to afford to use the gene editing therapies. 

“To my mind, the biggest and most pressing issues are accessibility and affordability, and those aren’t unique to genome editing at all. I worry about things like major medical advances that never see the light of day because they don’t fit someone’s business model or ones that can only be accessed by a certain privileged group of people,” Doudna said. 

This year, researchers from Editas Medicine became the first in the US to use the CRISPR-Cas9 tool in humans to try to treat a rare form of blindness, and so far, the preliminary findings show promise of safety and efficacy in using the CRISPR tool as a potential treatment.

“The eye is interesting in part because it has less immune reactivity than other tissues, it’s small, and self-contained, so the thinking is that there is a lower risk of unwanted edits or immune responses,” Doudna said. 

Doudna said researchers are also very close to a CRISPR-based cure for sickle cell disease, but that it is not affordable and they won’t be able to scale up the treatment to meet the need around the world without more refinements that bring the cost down. 

“Over the next decade, we’ll see a number of new CRISPR-based cures. There’s more and more every year entering clinical trials but broad acceptance and availability will take additional time,” said Doudna. 

“Nearly everyone has a mobile phone today but in the early days only a few people could afford them. I think a lot about how we can compress that time when it comes to CRISPR-based cures. You can wait for a mobile phone but people around the world don’t have that luxury when it comes to their health,” said Doudna. 

Alexa can be reached at [email protected].

A+
a-

In The News

Health

Voting

Health

January 24, 2022
by Tom Ramstack
Sarah Palin Diagnosed With COVID Before New York Times Lawsuit Trial

NEW YORK — Former vice-presidential candidate Sarah Palin was diagnosed with COVID-19 this weekend, which delayed her defamation lawsuit against... Read More

NEW YORK — Former vice-presidential candidate Sarah Palin was diagnosed with COVID-19 this weekend, which delayed her defamation lawsuit against The New York Times that was scheduled for a trial beginning Monday. Palin reported her illness to Judge Jed Rakoff of the U.S. District Court in... Read More

January 24, 2022
by Reece Nations
Political Polarization Impeded Public Support for COVID-19 Response

SAN ANTONIO — Public sentiment regarding COVID-19 mitigation strategies was undermined by polarization from political actors around the globe, according... Read More

SAN ANTONIO — Public sentiment regarding COVID-19 mitigation strategies was undermined by polarization from political actors around the globe, according to new research published in the Proceedings of the National Academy of Sciences. Researchers from around the world contributed to the study which examined the effects... Read More

January 24, 2022
by Tom Ramstack
FBI Cracks Down on Organizations Accused of Cashing In on the Pandemic

WASHINGTON —  The FBI is investigating an Illinois company that has received $124 million from the federal government for COVID-19... Read More

WASHINGTON —  The FBI is investigating an Illinois company that has received $124 million from the federal government for COVID-19 testing after reports the owners were using part of the money for lavish lifestyles. FBI agents raided the headquarters in Rolling Meadows, Illinois, Saturday. It opened... Read More

New Conservative Target: Race as Factor in COVID Treatment

MADISON, Wis. (AP) — Some conservatives are taking aim at policies that allow doctors to consider race as a risk... Read More

MADISON, Wis. (AP) — Some conservatives are taking aim at policies that allow doctors to consider race as a risk factor when allocating scarce COVID-19 treatments, saying the protocols discriminate against white people. The wave of infections brought on by the omicron variant and a shortage of... Read More

China Tests 2M in Beijing, Lifts COVID Lockdown in Xi’an

BEIJING (AP) — A cluster of COVID-19 cases in Beijing has prompted authorities to test millions and impose new measures... Read More

BEIJING (AP) — A cluster of COVID-19 cases in Beijing has prompted authorities to test millions and impose new measures two weeks ahead of the opening of the Winter Olympics, even as the city of Xi'an in north-central China lifted on Monday a monthlong lockdown that... Read More

US Jobless Claims Rise to 286,000, Highest Since October

WASHINGTON (AP) — The number of Americans applying for unemployment benefits rose to the highest level in three months as... Read More

WASHINGTON (AP) — The number of Americans applying for unemployment benefits rose to the highest level in three months as the fast-spreading omicron variant continued to disrupt the job market. Jobless claims rose for the third straight week — by 55,000 to 286,000, highest since mid-October,... Read More

News From The Well
scroll top