Israel aiming to enable unprecedented human DNA edits, to help cure illnesses
Genome editing is promising but imprecise, so Israel is setting up a consortium to make technology more exact; hopes to pave the way for pinpointed tweaks to human DNA
An illustrative image of DNA editing (CIPhotos; iStock by Getty Images)
Israel is setting up a task force aimed at bringing a new level
of accuracy to genome editing, in the hope it will pave the way to cures
for various medical conditions.
The state-run Israel Innovation Authority (IIA) has allocated NIS 36 million ($10 million) for the country’s academic institutions and several companies to advance CRISPR, a technology for making edits to the genome.
The consortium, which will work on genome editing for humans, as well as agriculture and fish, will run for 18 months, after which funding may be renewed.
There is widespread excitement in scientific circles about genome editing, after CRISPR was administered, for the first time, inside a human body last month, in an attempt to treat genetic condition that causes blindness.
Genome editing, which involves making changes to DNA, normally to address a health problem, is thought to have potential for common conditions like cancer and blood disorders, but also for rare genetic illnesses for which medicines aren’t developed.
“Israel will be one of the leaders in the field,” IIA vice president
Aviv Zeevi told The Times of Israel, saying this hope is driving the
establishment of the new CRISPR-IL consortium. “We’ll have some of the
most accurate tools.”
Zeevi, who heads IIA’s Technological Infrastructure Division, said that the new consortium will deploy artificial intelligence to try to overcome one of the major hurdles of genome editing — its accuracy levels.
“We need to be able to target the specific part of the genome we want to fix, and the difficulty is that accuracy of existing tools is low,” he said.
He stated that members of the consortium will research ways to boost accuracy by developing ways of guiding the tools that make genome edits — “tools that are like scissors which cut the specific part of the sequence and replace it” — to the precise place where they are needed.
More controversially, in 2018, Chinese researcher He Jiankui claimed
to have produced the world’s first gene-edited babies. He was sentenced to three years in prison.
Israel has strict laws governing scientific endeavors, and Zeevi said
that no research involving humans will take place without approval by an
ethical committee.
The same technology will advance genome editing for humans, agriculture and fish, Zeevi predicted.
He admitted that Israel is taking a leap into the unknown. “We’re taking a big risk here,” he said. “It’s not a very standard field in which we’re investing. We know we’re going into a field that’s very high risk, and we’re trying to create a new economic impact on the field. We’re trying to identify an interdisciplinary niche in which we’ll be able to lead.”
The state-run Israel Innovation Authority (IIA) has allocated NIS 36 million ($10 million) for the country’s academic institutions and several companies to advance CRISPR, a technology for making edits to the genome.
The consortium, which will work on genome editing for humans, as well as agriculture and fish, will run for 18 months, after which funding may be renewed.
There is widespread excitement in scientific circles about genome editing, after CRISPR was administered, for the first time, inside a human body last month, in an attempt to treat genetic condition that causes blindness.
Genome editing, which involves making changes to DNA, normally to address a health problem, is thought to have potential for common conditions like cancer and blood disorders, but also for rare genetic illnesses for which medicines aren’t developed.
Genome editing is already used in agriculture and research, and it is widely hoped that successes with animals — like reducing the severity of genetic hearing loss in mice — will provide the knowledge that scientists need to advance human health.
Aviv Zeevi, vice president of the Israel
Innovation Authority and head of its Technological Infrastructure
Division (courtesy of the Israel Innovation Authority)
Zeevi, who heads IIA’s Technological Infrastructure Division, said that the new consortium will deploy artificial intelligence to try to overcome one of the major hurdles of genome editing — its accuracy levels.
“We need to be able to target the specific part of the genome we want to fix, and the difficulty is that accuracy of existing tools is low,” he said.
He stated that members of the consortium will research ways to boost accuracy by developing ways of guiding the tools that make genome edits — “tools that are like scissors which cut the specific part of the sequence and replace it” — to the precise place where they are needed.
Zeevi said: “We will train algorithms to
know where to perform the edit and get to a higher accuracy level.
Right now, the success rate for genome edits is around 10% and we are
hoping to get to 70% within three years.”
He said that the algorithms will both
pinpoint the right targets for edits, and reduce risk of edits having
unwelcome knock-on effects on other parts of the genome — a major
concern that holds back human DNA editing.
To date, genome editing in humans has been
limited, though there have been some successes. Last year, the
University of Pennsylvania announced that it had conducted the first
clinical trial to edit a cancer patient’s immune cells using CRISPR
technology and then return the cells to the patient. It concluded that
the method is safe and feasible.
In this Oct. 10, 2018 photo, He Jiankui
speaks during an interview at a laboratory in Shenzhen in southern
China’s Guangdong province. (AP/Mark Schiefelbein)
The same technology will advance genome editing for humans, agriculture and fish, Zeevi predicted.
He admitted that Israel is taking a leap into the unknown. “We’re taking a big risk here,” he said. “It’s not a very standard field in which we’re investing. We know we’re going into a field that’s very high risk, and we’re trying to create a new economic impact on the field. We’re trying to identify an interdisciplinary niche in which we’ll be able to lead.”
This consortium is part of the IIA’s
Bio-convergence Initiative, which brings together engineering, AI,
physics, computation, nanotechnology, material science and advanced
genetic engineering to address needs in health and other industries,
such as agriculture and energy.
The consortium includes six academic institutions, two hospitals and eight companies, including plant genomics companies Nrgene and Evogene.
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The CRISPR-Cas9 system can be programmed to modulate the population of any bacterial species by targeting clinical genotypes or epidemiological isolates. It can selectively enable the beneficial bacterial species over the harmful ones by eliminating pathogen, which gives it an advantage over broad-spectrum antibiotics.[38]
Antiviral applications for therapies targeting human viruses such as HIV, herpes, and hepatitis B virus are under research.
CRISPR demonstrated the ability to target and eliminate endogenous retroviruses, which reduces the risk of transmitting diseases and reduces immune barriers.[36] Eliminating these problems improves donor organ function, which brings this application closer to a reality.
David Andrew Sinclair notes that "the new technologies with genome editing will allow it to be used on individuals (...) to have (...) healthier children" – designer babies.[73]
The American National Academy of Sciences and National Academy of Medicine issued a report in February 2017 giving qualified support to human genome editing.[76] They recommended that clinical trials for genome editing might one day be permitted once answers have been found to safety and efficiency problems "but only for serious conditions under stringent oversight."[77]
its deliberate or unintentional misuse might lead to far-reaching economic and national security implications.[78][79][80] For instance technologies such as CRISPR could be used to make "killer mosquitoes" that cause plagues that wipe out staple crops.
will allow amateurs – or "biohackers" – to perform their own experiments, posing a potential risk from the release of genetically modified bugs.
risks and benefits of modifying a person's genome – and having those changes pass on to future generations – are so complex that they demand urgent ethical scrutiny. Such modifications might have unintended consequences which could harm not only the child, but also their future children,
the provided sensitive information could lead to the manufacture of biological weapons by potential bioterrorists who might use the knowledge to create vaccine resistant strains of other pox viruses, such as smallpox, that could affect humans
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human DNA edits, to help cure illnesses
Genome editing
In the near future the new CRISPR system will also be able to eradicate diseases and conditions that humans are predisposed for. With this new technology scientists will be able to take the genes of a human sperm cell and egg, and replace the genes that activate cancer or other abnormal or unwanted defects. This will take the stress off of parents worrying about having a child and them not being able to live it like a normal child should. After just one generation of this process, the entire future of the human race would never have to worry about the problems of deformities or predisposed conditions.[66]Eradicating diseases
The CRISPR-Cas9 system can be programmed to modulate the population of any bacterial species by targeting clinical genotypes or epidemiological isolates. It can selectively enable the beneficial bacterial species over the harmful ones by eliminating pathogen, which gives it an advantage over broad-spectrum antibiotics.[38]
Antiviral applications for therapies targeting human viruses such as HIV, herpes, and hepatitis B virus are under research.
Prospects and limitations
Genome editing occurs also as a natural process without artificial genetic engineering. The agents that are competent to edit genetic codes are viruses or subviral RNA-agents.CRISPR demonstrated the ability to target and eliminate endogenous retroviruses, which reduces the risk of transmitting diseases and reduces immune barriers.[36] Eliminating these problems improves donor organ function, which brings this application closer to a reality.
Human enhancement
David Andrew Sinclair notes that "the new technologies with genome editing will allow it to be used on individuals (...) to have (...) healthier children" – designer babies.[73]
The American National Academy of Sciences and National Academy of Medicine issued a report in February 2017 giving qualified support to human genome editing.[76] They recommended that clinical trials for genome editing might one day be permitted once answers have been found to safety and efficiency problems "but only for serious conditions under stringent oversight."[77]
Risks
its deliberate or unintentional misuse might lead to far-reaching economic and national security implications.[78][79][80] For instance technologies such as CRISPR could be used to make "killer mosquitoes" that cause plagues that wipe out staple crops.
will allow amateurs – or "biohackers" – to perform their own experiments, posing a potential risk from the release of genetically modified bugs.
risks and benefits of modifying a person's genome – and having those changes pass on to future generations – are so complex that they demand urgent ethical scrutiny. Such modifications might have unintended consequences which could harm not only the child, but also their future children,
the provided sensitive information could lead to the manufacture of biological weapons by potential bioterrorists who might use the knowledge to create vaccine resistant strains of other pox viruses, such as smallpox, that could affect humans
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