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How human gene editing is moving on after the CRISPR baby scandal
By Katie Hunt, CNN Published 12:46 PM EST, Thu March 9, 2023
For most of her life, Victoria Gray, a 37-year-old mother of four from Mississippi, had experienced excruciating bouts of pain.
Born with the blood disorder sickle cell disease, lengthy hospital stays and debilitating fatigue disrupted her childhood, forcing her to quit pursuing a college nursing degree and take potent and addictive painkillers.
“The pain I would feel in my body was like being struck by lightning and hit by a freight train all at once,” she said this week at the Third International Summit on Human Genome Editing in London.
In 2019, she received an experimental treatment for the inherited disease that used the gene-editing technique CRISPR-Cas9, which allowed doctors to make very precise changes to her DNA. While the procedure itself was grueling and took seven to eight months to fully recover from, she said it has transformed her life.
“The feeling is amazing. I really feel that I’m cured now,” Gray said. “Because I no longer have to face the battles that I faced on a day-to-day (basis). I came from having to have an in-home caregiver to help me take baths, clean my house and care for my children. Now I do all those things on my own.”
She’s now able to enjoy a life she once felt was passing her by. She holds down a full-time job as a Walmart cashier, and she’s able to attend her children’s football games and cheerleading events and enjoy family outings. “The life I felt I was just existing in I’m now thriving in,” she said.
Gray shared her experience with doctors, scientists, patient advocates and bioethicists who gathered in London for the human genome editing summit, at which participants reported on advances made in the field and debated the thorny ethical issues posed by the cutting-edge technology.
“I’m here really to be a light because there’s mixed feelings about gene editing. And I think people can see the positive result of it. You know that a person who was once suffering in life, was miserable, now is able to be a part of life and enjoy it,” Gray told CNN.
Baby scandal lingers
Gray’s uplifting story, which received a standing ovation from the audience, stood in contrast to a presentation made the last time the conference was held, in Hong Kong in 2018, when Chinese doctor He Jiankui stunned his peers and the world with the revelation that he had created the world’s first gene-edited babies.
The two girls grew from embryos He had modified using CRISPR-Cas9, which he said would make them resistant to HIV. His work was widely condemned by the scientific community, which decried the experiment as medically unnecessary and ethically irresponsible. He received a three-year jail sentence in 2019.
Questions about the baby scandal still linger more than four years later, and after being recently released from prison, He is reportedly seeking to continue his work. China has tightened its regulation of experimental biomedical research since 2018, but it hasn’t gone far enough, said Joy Zhang, a medical sociologist at the University of Kent in the United Kingdom.
“Ethical governance in practice is still confined to traditional medical, scientific, as well as educational, establishments. The new measures fail to directly address how privately funded research and other … ventures will be monitored,” Zhang said at the conference.
Ethically questionable experimental research isn’t an issue confined to China, said Robin Lovell-Badge, head of the Laboratory of Stem Cell Biology and Developmental Genetics at the Francis Crick Institute in London, who chaired the 2018 Hong Kong conference session in which He attempted to defend his work.
“(He Jiankui) is not the only concern in this area. One of our big concerns I always have is the possibility that there will be rogue companies, rogue scientists setting up to do genome editing in an inappropriate way,” Lovell-Badge said on Monday at the conference.
Hopes and fears around gene editing
While the CRISPR baby scandal tarnished the technology’s reputation, CRISPR-Cas9 and related techniques have made a major impact on biomedical research, and two scientists behind the tool — Emmanuelle Charpentier and Jennifer A. Doudna — won a Nobel prize for their work in 2020.
“Clinical trial results demonstrate that CRISPR is safe, and it’s effective for treating and curing human disease — an extraordinary advance given the technology is only 10 years old,” Doudna said at the conference in a video address. “It’s important with a powerful technology like this to grapple with the challenges of responsible use.”
In addition to the sickle cell trial that includes Gray, clinical trials are also underway to test the safety of gene editing in treating several other conditions, including a related blood disorder called beta thalassemia; leber congenital amaurosis, which is a form of inherited childhood blindness; blood cancers such as leukemia and lymphoma; type 1 diabetes; and HIV/AIDS.
DNA acts as a instruction manual for life on our planet, and CRISPR-Cas9 can target sites in plant and animal cells using guide RNA to get the Cas-9 enzyme to a more precise spot on a strand of DNA. This allows scientists to change DNA by knocking out a particular gene or inserting new genetic material at a predetermined site in the strand.
People with sickle cell disease have abnormal hemoglobin in red blood cells that can cause them to get hard and sticky, clogging blood flow in small vessels.
In the trial that Gray was part of, doctors increased the production of a different kind of hemoglobin, known as fetal hemoglobin, which makes it harder for cells to sickle and stick together. The process is invasive and involves removing premature cells from the bone marrow and modifying them — by using CRISPR-Cas9 in the lab — to eventually produce fetal hemoglobin. The patient has to undergo a round of chemotherapy before receiving the gene-edited cells to ensure the body doesn’t reject them.
The conference also shed light on new, more sophisticated gene-editing techniques, such as prime editing and base editing, which recently was used to modify immune cells and successfully treat a teen with treatment-resistant leukemia.
These next generation techniques will allow humans “to have some say in the sequence of our genomes so we are no longer so beholden to the misspellings in our DNA,” said David Liu, the Richard Merkin professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard University.
The gene therapy trials currently underway involve treating people who were born with a certain disease or condition by altering non-reproductive cells in what’s known as somatic gene editing.
The next frontier — many would say red line — is heritable gene editing: altering the genetic material in human sperm, eggs or embryos so that it can be safely passed onto the next generation. The goal would be to prevent babies from inheriting genetic diseases.
“It’s a very different set of ethical trade-offs when you’re not a treating disease in an existing individual but you’re in fact preventing an individual yet to be born from suffering from a disease. That’s a very different set of considerations,” said George Daley, Caroline Shields Walker Professor of Medicine and dean of the faculty of medicine at Harvard Medical School.
In a statement released at the end of the conference, the organizers said “heritable human genome editing remains unacceptable at this time.”
They added that public discussion and policy debates should continue and were important for resolving whether this technology should be used.
CRISPR access and equity
The hope offered by gene therapy is creating fresh ethical storms — primarily over who gets access to such treatments. The therapy Gray received, which is expected to soon receive regulatory approval, is likely to cost more than $2 million per person, putting it out of reach for many who need it in the United States and in low-income countries.
“If we want to be serious about equitable access to these kinds of therapies, we have to start talking early on about ways to develop them and make them available and make them cost effective and sustainable,” said Alta Charo, the Warren P. Knowles Professor Emerita of Law and Bioethics at the University of Wisconsin at Madison.
Researchers want to develop CRISPR therapies that can be delivered though an injection rather than the chemotherapy and invasive bone marrow transplant Gray went through.
Worldwide, more than 300,000 children are born with sickle cell disease every year, over 75% of whom live in sub-Saharan Africa, where screening programs and treatment options are limited.
Even relatively affordable drugs to treat sickle cell disease, such as hydroxyurea, don’t reach everyone who needs them in India, said Gautam Dongre, the secretary of the National Alliance of Sickle Cell Organizations in India and father of two children with sickle cell disease.
“After 40 years if these drugs aren’t reachable for the common people, then what about gene therapy?” Dongre asked at the conference.
Julie Makani, an associate professor in the department of haematology and blood transfusion at Muhimbili University of Health and Allied Sciences in Tanzania, said more genomic research should take place in Africa.
“The ultimate thing for me, particularly as a physician scientist, is not just discovery, but also seeing the application of knowledge…into (an) improvement in health,” Makani said.
But how can they call on him (Jesus Christ) to save them unless they believe in him (Jesus Christ)? And how can they believe in him (Jesus Christ) if they have never heard about him (Jesus Christ)? And how can they hear about him (Jesus Christ) unless someone tells them?” —Romans 10:14
In His Service,
Night Watchman Ministries
Make Your Decision for Christ NOW!!!!!!! Time is Up!!!!!!!
Jesus Christ’s Offer of Salvation:
The ABCs of Salvation through Jesus Christ (the Lamb)
A. Admit/Acknowledge/Accept that you are sinner. Ask God’s forgiveness and repent of your sins.
. . . “For all have sinned, and come short of the glory of God.” (Romans 3:23).
. . . “As it is written, There is none righteous, no, not one.” (Romans 3:10).
. . . “If we say that we have no sin, we deceive ourselves, and the truth is not in us.” (1 John 1:8).
B. Believe Jesus is Lord. Believe that Jesus Christ is who He claimed to be; that He was both fully God and fully man and that we are saved through His death, burial, and resurrection. Put your trust in Him as your only hope of salvation. Become a son or daughter of God by receiving Christ.
. . . “That whosoever believeth in him should not perish, but have eternal life. For God so loved the world, that he gave his only begotten Son, that whosoever believeth in him should not perish, but have everlasting life. For God sent not his son into the world to condemn the world; but that the world through him might be saved. (John 3:15-17). For whosoever shall call upon the name of the Lord shall be saved.” (Romans 10:13).
C. Call upon His name, Confess with your heart and with your lips that Jesus is your Lord and Savior.
. . . “That if thou shalt confess with thy mouth the Lord Jesus, and shalt believe in thine heart that God hath raised him from the dead, thou shalt be saved. For with the heart man believeth unto righteousness; and with the mouth confession is made unto salvation.” (Romans 10:9-10).
. . . “If we say that we have no sin, we deceive ourselves, and the truth is not in us. If we confess our sins, he is faithful and just to forgive us our sins, and to cleanse us from all unrighteousness. If we say that we have not sinned, we make him a liar, and his word is not in us.” (John 1:8-10).
. . . “And he is the propitiation for our sins: and not for ours only, but also for the sins of the whole world. (John 2:2).
. . . “In this was manifested the love of god toward us, because that God sent his only begotten Son into the world, that we might live through him. And we have seen and do testify that the Father sent the Son to be the Saviour of the world. Whosoever shall confess that Jesus is the Son of God, God dwelleth in him, and he in God.” (1 John 4:9, 14-15).
. . . “But God commendeth his love toward us, in that, while we were yet sinners, Christ died for us. Much more then, being now justified by his blood, we shall be saved from wrath through him. For if, when we were enemies, we were reconciled to God by the death of his Son, much more, being reconciled, we shall be saved by his life.” (Romans 5:8-10).
. . . “For the wages of sin is death; but the gift of God is eternal life through Jesus Christ our Lord.” (Romans 6:23).
. . . “Jesus saith unto them, I am the way, the truth, and the life, no man cometh unto the Father, but by me.” (John 14:6).
. . . “For I am not ashamed of the gospel of Christ: for it is the power of God unto salvation to everyone that believeth.” (Romans 1:16).
. . . “Neither is there salvation in any other: for there is none other name under heaven given among men, whereby we must be saved.” (Acts: 4:12).
. . . “Who will have all men to be saved, and to come unto the knowledge of the truth for there is one God, and one mediator between God and men, the man Christ Jesus.” (1 Timothy 2:4-6).
. . . “For God did not appoint us to suffer wrath but to receive salvation through our Lord Jesus Christ.” (1 Thessalonians 5:9).
. . . “But as many as received him, to them gave the power to become the sons of God, even to them that believe on his name.” (John 1:12).
True Church / Bride of Christ Spared from God’s Wrath:
Romans 5:8-10. “But God commendeth his love toward us, in that, while we were yet sinners, Christ died for us. Much more then, being now justified by his blood, we shall be saved from wrath through him. For if, when we were enemies, we were reconciled to God by the death of his Son, much more, being reconciled, we shall be saved by his life.”
Romans 12:19. Dearly beloved, avenge not yourselves, but rather give place unto wrath: for it is written, Vengeance is mine; I will repay, saith the Lord.
1 Thessalonians 1:10. And to wait for his Son from heaven, whom he raised from the dead, even Jesus, which delivered us from the wrath to come.
1 Thessalonians 5:9. For God hath not appointed us to wrath, but to obtain salvation by our Lord Jesus Christ,
Romans 8:35. Who shall separate us from the love of Christ? shall tribulation, or distress, or persecution, or famine, or nakedness, or peril, or sword?
Jeremiah 30:7. Alas! for that day is great, so that none is like it: it is even the time of Jacob’s trouble, but he shall be saved out of it.
Revelation 3:10 Because thou hast kept the word of my patience, I also will keep thee from the hour of temptation, which shall come upon all the world, to try them that dwell upon the earth.