2019: the year gene therapy came of age

Yahoo – AFP, Ivan Couronne, December 26, 2019

Victoria Gray's blood was drawn so that doctors could get to the cause of her illness
-- stem cells from her bone marrow that were making deformed red blood cells (AFP Photo)

Washington (AFP) - In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

DNA from Gray's stem cells was modified at a Scottish laboratory, using Crispr/Cas9 -- 

pronounced "Crisper" -- a new tool informally known as molecular "scissors" (AFP 

Photo/GERARD JULIEN)

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Crispr made gene editing more widely accessible - but has given new impetus to the 

perennial debate over the wisdom of humanity manipulating life (AFP Photo/ERIC 

PIERMONT)

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and an inherited blindness.

Scientific director of the Telethon Serge Braun poses for a photograph after a press 

conference to present the Telethon 2018, the annual fundraising for medical research, 

at the Myology Institute in Paris on December 3, 2018 (AFP Photo)

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

French geneticist Emmanuelle Charpentier, one of Crispr's inventors, doesn't believe 

in the more dystopian scenarios that have been predicted for gene therapy (AFP Photo/

ANDER GILLENEA)

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his excommunication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

Chinese scientist He Jiankui, pictured in 2018, used Crispr to create what he called 

the first gene-edited humans (AFP Photo/Anthony WALLACE)

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

A picture taken at the French National Institute of Agricultural Research (INRA) in 

Maison-Alfort, on July 20, 2016 shows a tick, whose bite can transmit the Lyme 

disease (AFP Photo/BERTRAND GUAY)

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

Radboud research offers new hope to MS sufferers, identifies key genes

DutchNews, November 18, 2019

Photo: Depositphotos.com

Scientists at Radboud University teaching hospital in Nijmegen say they have discovered multiple dna variants which indicate an increased risk of developing Muscular Sclerosis. 

The discovery will allow researchers to carry out more targeted research into the disease, according to MS charity Nationaal MS Fonds.  MS is a disease of the central nervous system that disrupts the flow of information within the brain, and between the brain and body. 

The most important of the Radboud discoveries focuses on the MBP gene, which produces a protein that forms a protective layer around the nerves. In MS patients, antibodies stop the protein being made, leaving the nerve unprotected. 

This, in turn, leaves the nerves unable to send signals to the brain, leading to paralysis. 

The scientists now want to use MS sufferers’ skin cells to grow stem and nerve cells to look for new remedies. 

‘We want to use some of the genes which have been found in a functional study to test at cell level how the genetic defects work,’ research association Geert Poelmans said. ‘The results of this follow-up research will focus on developing long-term new treatments to counteract MS’ 

Scientists believe that a combination of environmental and genetic factors contribute to the risk of developing MS. Some 17,000 people in the Netherlands currently have MS, with 800 new cases being diagnosed every year. 

The new discoveries were made comparing the dna of MS sufferers and their healthy family members.

Related Articles:

Human-like neural activity detected in lab grown mini brains

Human muscle tissue grown from stem cells: US study

"The Addictive Old Energy" + "Timing", US (3), August 12-13, 2017(Kryon Channelling by Lee Carroll)  (The Addictive Old Energy - Text version)

"... Is He Alone? How About You?

What kind of addiction does the old energy have for you? Are you continuing to do things that you think are absolutely correct, working with perceptions that have been given to you in an old energy that you are still wallowing in? Of course, reading this you will say, "Not me." Remember the Korean leader. He still thinks he is right and never saw it. How invested in struggle are you? I have some news. You can't do it for long. First, you lose friends. You can't claim to be one thing and be another anymore.

So let's start extending this teaching. If the darkness is so attractive, is it affecting anything else? The answer? It is absolutely affecting everything else. There's an entire industry we have told you about that will eventually fail because it has been developed in an older energy, worked in an older energy, and has no integrity. The industry is Big Pharma. We've told you that its fall is imminent and the attributes will come from the ones within it, who will expose it. When this actually happens is up to you, since this is free choice of the developing consciousness of your country. However, let's look for a moment, not at the event, but what it may then lead to.

Big Pharma is invested in an old idea that designer chemistry is the answer to healing. This idea has been prevalent for decades and many feel that it is the highest technology available. However, instead, and in the light, there starts to appear another idea entirely: The Human body has an unrealized awareness and it is actually the master healer! The Human body is designed to self-correct. So, therefore, don't put foreign chemistry into it, but instead find out how to give it elegant, specific instruction sets to heal itself. Make them high-technology instruction sets to inform it to heal itself!

This is homeopathy expanded, you might say. There are processes coming that understand that this Human body of yours is so much better than any chemist has ever realized, and the new energy is starting to expose this fact. The answer to healing is not chemistry. The answer to health and healing is by addressing the "smart part" of the Human body, giving it instructions to utilize former unknown systems that are ready to create spontaneous remission and beyond.

Remember my channelling given on the "youthing template" or the fact that you all have dormant "genesis cells" within your body (cells that have the full body blueprint and a source for very young stem cells)? These both were expositions of systems that are smart and ready to create new cells and healing in ways that will seem miraculous! But they need to be "instructed". 

Funding Will Trail for Awhile

How much money do you think is invested in the old ways? Right now, there is an industry of investment that is going to fail. So, do you think all this money will then be invested into the new methods? The answer is NO, not right away. Just like the North Korean leader's blindness, the old ways are just too addicting. "Keep it the same," they will yell. "The fall is temporary," they will say. "It always worked before, and it always will." Many investors will lose fortunes. The dysfunction is already being seen clearly. When you turn on your media at night, much of what you hear in the commercial breaks is how the chemistry didn't work! Now you get a chance to join others and sue the pharma companies for bad chemistry! You think something is beginning to break? It is. You see, they are already wallowing in something that's going to make them fail. They are advertising how broken the very system is. Both big pharma and their investors don't see the light coming yet, even though it is becoming dysfunctional before their eyes.  ..."

"The Innate Revealed" (3) - Nov 22/23, 2014 (Kryon Channelling by Lee Carroll) - (Text version)

“… Survival Programming

Whereas the Human brain is programmed for corporeal survival, Innate is programmed for spiritual survival. You might ask, "What is the difference?" Corporeal survival is your ability to get away from the tiger chasing you and find food and shelter. It gives you the logic to figure out these things and to stay alive. Spiritual survival is a very different thing, and it's very esoteric. It's a much larger kind of survival. It pushes humanity's spiritual evolution and provides a higher working level of your DNA. Let me explain.

Innate knows what the ancients knew. It knows the grand plan; remember, it's connected to the Higher-Self. It knows how long humanity has on this planet before it will no longer be able to make a decision toward planetary graduation. Innate knows that; your DNA knows that. What is the prime directive of Innate? It is to do everything it can for you, giving you multiple chances at free choice, allowing an awakening to take place, therefore, allowing humanity to go across the bridge of survival and move into an ascended planet status. That is the prime directive.

Spiritual survival represents the potential evolution of Earth, and everything is designed around that. Innate is designed to push you forward in any way possible within the free choice system into spiritual awareness. That is what Innate is for. Do you see the grand plan in all this? You have a built-in guidance system to help you navigate your potential.

In this process, Innate also crosses the bridge with corporeal chemistry in very different ways. Innate is responsible for spontaneous remission. Now you know where that comes from. Did you really think that came out of the brain? How can a Human have a disease disappear overnight? How is it possible that the corporeal body can cleanse itself of something so unbalanced overnight? How can tissue grow in a greatly accelerated rate to cause healing almost overnight? I have just given you things that hospitals have seen over and over. They have X-rayed them and verified them and chemically tested them. There is no way, they might say, that this could normally happen. For those who are spiritual, it has to be a miracle! For the science minded, it remains unexplainable. The beauty of this is that it is, indeed, explainable. It's the miracle of Innate! It's your own body keeping you alive. It's a profound, miraculous back-up system.

That's the power that you have. When Innate finally starts to build the bridge to the corporeal self, the Human Being as you know it today will disappear. The one who takes its place will have a much longer life, able to repair itself and even grow limbs back. This is how it was designed, dear ones, and this should start to make sense to you. …”

"The Mysterious Innate" – Aug 31, 2013 (Kryon Channelling by Lee Carroll) - (Text version)

“.. Mining the Akash - The Role of Innate

What I'm going to tell next, we have given you before. However, I wish to explain it better in this communication. Your innate is also a governor of what you personally need. Let me explain: Turn a page with me. We have talked in the past about mining the Akash. So let us open that door for a moment. Is it possible, dear one, that you could go into the vast storehouse of the attributes of your past lives and pick out something that you need today? The answer is yes. It's called mining the Akash, and it is a staple of the Kryon teaching. However, the way it's accomplished by the individual Human is really difficult to describe. It's a personal intuitive process. However, one attribute of it that is often misunderstood is that permission from the innate is needed. This is because the innate smart body knows what you need. It is then the governor, or the filter, giving you permission regarding what can be pulled out of your Akash and used. It will say no to frivolous things and yes to the things that will allow you to cure yourself of disease, live longer, and increase the efficiency of your DNA. It's a smart body regulator.

Innate doesn't care how you look. It cares about your health. If you want to go into your Akash and pull out something that you need for survival, it's right there with you. If you want to make clearer skin, it won't respond. Do you see what I mean? It's the governor for mining the Akash. By the way, the Akash is very ready to be used for this. [Kryon wink]

You have earned what you've lived, dear ones, and it's very available. It lies in a quantum soup of lifetime information that is ready to be used. If you have a disease in your body right now, and perhaps you want to mine the Akash in a way that will create healing, you can. Like all nonlinear things, it requires a new, mature consciousness and practice to work. But the body is ready for you to "go get" the cellular structure of a past life that had no disease. The blueprint for this resides within you since you earned it and lived it. The innate is responsible for spontaneous remission! Science has no idea how a Human does this. The innate can help you "go get" the things within your earned Akash and place them into your cellular structure. That's how smart it is! Imagine clearing disease so completely that there is no sign it was ever there? It happens all the time. Imagine being able to drop a chemical dependency overnight? It's exactly the same principle.

Did you ever sit in a meeting and something is said or a feeling can happen, and you get chills? What are they? You might say, "They are chills of validation." Guess where they came from? The innate! The innate has the ability to signal you the truth. The brain does not. In fact, your brain will often get in the way. It's the brain that contains your box of belief. It's the brain that contains the perception of who you are based on experience and memory. It's your 3D survival instrument. But the brain can't give truth. It can only give you what it perceives as the truth, based on logical, processed, computed, synaptic reasoning and only based on your past experience.

Your brain can tell you not to touch the stove because of what happened once before. But it can't give you chills of validation from information it does not have. The intellect wants you to believe that your brain is supreme. However, the brain is only one part of the body system and not a very intuitive part. It is the innate that knows the truth. The innate is connected to the quantum parts of your DNA and, therefore, also knows of your spirituality and the truth of God inside. …”

"The Quantum Factor" – Apr 10, 2011 (Kryon channeled by Lee Carroll) (Subjects: Galaxies, Universe, Intelligent design, Benevolent design, Aliens, Nikola Tesla (Quantum energy), Inter-Planetary Travel, DNA, Genes, Stem Cells, Cells, Rejuvenation, Shift of Human Consciousness, Spontaneous Remission, Religion, Dictators, Africa, China, Nuclear Power, Sustainable Development, Animals, Global Unity.. etc.) - (Text Version)

“… Cell Division - a static process?

Let me take you to the cellular division process. We've said this before, but you need to hear this to understand how it works. A cell is ready to divide. The Human body is designed to rejuvenate... all tissue. You've been told that there's some tissue that does not rejuvenate, but that is incorrect. It all rejuvenates at different speeds at different times and in different ways. It rejuvenates. So now you know that the Human body is designed to live a long time. Unfortunately, the energy that you have created on this planet and what you've gone through, has beat it up. You don't live much more than 80 years. That was not the design.

The Biblical personalities were sometimes prophets and sometimes masters and sometimes just there... and lived for hundreds of years. Did they really? Or perhaps this is that just a metaphor? Did they get that right in the Bible without a error in transcription? I'm going to tell you the truth. It's very accurate. Thousands of years ago you lived a very long time, Lemurian. If you knew your lifespan, you'd gasp. But not anymore. Instructions have been given over time to DNA, literally, by the energy of the planet... en energy that you have created through consciousness.

A cell divides. Right before it divides, it needs the blueprint to clone itself. The blueprint is available from the stem cell. The stem cell gets its information from the quantum part of the DNA, which has never changed since you were born. It's remained static, since nothing has ever changed it... and the fact that you don't believe it's changeable and have just accepted aging. There's not a conscious effort to do anything with it, and it just lays there like it always did.

The diving cell "talks" to the stem cell and says, "Do the same thing you always did? Change anything?" And the stem cell talks to the cell that is dividing, saying, "Make another one just the same." Then you rejuvenate just like the last one, accepting everything you received when you were born.  …”

Human-like neural activity detected in lab grown mini brains

Yahoo – AFP, Issam AHMED, August 29, 2019

A cross-section of a brain organoid, showing the initial formation of a cortical
plate, with each color marking a different type of brain cell (AFP Photo/HO)

Washington (AFP) - Scientists reported Thursday they had picked up human-like electrical activity in lab-grown brains for the first time, paving the way to model neurological conditions and answer fundamental questions on how our gray matter develops.

It's not clear whether the pea-sized brains are conscious: the team behind the breakthrough suspect they're not because the activity resembles that of preterm babies, but they cannot say for certain, opening up a new ethical dimension to this area of research.

So-called "cerebral organoids" derived from adult stem cells have been around for a decade or so but have never previously developed functional neural networks.

"If you had asked me five years ago 'Would you think that a brain organoid would ever have a sophisticated network able to generate a brain oscillation?' I would say no," Alysson Muotri, a biologist at the University of California San Diego, told AFP.

A paper published by Muotri and his colleagues in the journal Cell Press said that two factors were responsible for the breakthrough. The first was a better procedure to grow stem cells, including optimizing the culture medium formula.

The second was initially surprising, but also intuitive when the researchers thought about it: simply allowing the neurons adequate time to develop, just as babies' brains develop in the womb.

"The very early stages of human neurodevelopment are encoded in our genome," explained Muotri.

The team began to detect bursts of brain waves from organoids from about two months.

The signals were sparse at first and all at the same frequency, a pattern seen in very immature human brains. But as they grew, they produced waves at different frequencies, and the signals appeared more regularly, suggesting further development of their neural networks.

Researchers then compared the brain wave patterns with those of human brains in early development, by training a machine learning algorithm with the activity recorded from 39 prematurely born babies.

The program was successful in predicting how many weeks the organoids had been developing in their dishes, suggesting they shared a similar growth trajectory to brains in their natural setting.

Pea-size brain organoids at 10 months old; so-called "cerebral organoids" derived

from adult stem cells have been around for a decade or so but have never 

previously developed functional neural networks (AFP Photo/HO)

The point at which newborn babies attain consciousness, and the definition of consciousness itself, are both contested among scientists.

A French study in 2013 that looked at the brain activity of babies found they began to think about images of faces they were shown from the age of five months, appearing to store the visuals in their temporary "working memory" -- a faculty they linked to perceptual consciousness.

Applications and ethics

Earlier, less sophisticated organoids were used to model what happens when the brain is exposed to diseases like the Zika virus which causes physical malformations.

But there are also a host of neurological conditions such as autism, epilepsy, and even psychiatric conditions where problems arise from how the brain network is wired, not from malformations.

By creating brain organoids from the stem cells of individuals with these conditions, scientists could better model them and perhaps one day find cures.

They also hope to answer more fundamental questions. Muotri said the organoids development plateaued around nine to 10 months, for reasons that aren't yet clear.

"I'm curious about that. I wonder if it's because we don't have a vascularization system to allow the nutrients to get inside, or it could be that we are just lacking stimulation" in the form of sensory input.

He hopes to test both hypotheses.

About whether the brain organoids are conscious, Muotri said he suspects not because of its early stage of development.

"But if you ask me, 'How do you know?' I would say I have no evidence either way, because we don't even know how to detect consciousness in other systems," he said.

"As we get closer to the human brain, all of these ethical questions will appear," he admitted, proposing that the field be subject to agreed-upon limits and regulation.

Related Articles:

Human muscle tissue grown from stem cells: US study

"The Addictive Old Energy" + "Timing", US (3), August 12-13, 2017(Kryon Channelling by Lee Carroll)  (The Addictive Old Energy - Text version)

"... Is He Alone? How About You?

What kind of addiction does the old energy have for you? Are you continuing to do things that you think are absolutely correct, working with perceptions that have been given to you in an old energy that you are still wallowing in? Of course, reading this you will say, "Not me." Remember the Korean leader. He still thinks he is right and never saw it. How invested in struggle are you? I have some news. You can't do it for long. First, you lose friends. You can't claim to be one thing and be another anymore.

So let's start extending this teaching. If the darkness is so attractive, is it affecting anything else? The answer? It is absolutely affecting everything else. There's an entire industry we have told you about that will eventually fail because it has been developed in an older energy, worked in an older energy, and has no integrity. The industry is Big Pharma. We've told you that its fall is imminent and the attributes will come from the ones within it, who will expose it. When this actually happens is up to you, since this is free choice of the developing consciousness of your country. However, let's look for a moment, not at the event, but what it may then lead to.

Big Pharma is invested in an old idea that designer chemistry is the answer to healing. This idea has been prevalent for decades and many feel that it is the highest technology available. However, instead, and in the light, there starts to appear another idea entirely: The Human body has an unrealized awareness and it is actually the master healer! The Human body is designed to self-correct. So, therefore, don't put foreign chemistry into it, but instead find out how to give it elegant, specific instruction sets to heal itself. Make them high-technology instruction sets to inform it to heal itself!

This is homeopathy expanded, you might say. There are processes coming that understand that this Human body of yours is so much better than any chemist has ever realized, and the new energy is starting to expose this fact. The answer to healing is not chemistry. The answer to health and healing is by addressing the "smart part" of the Human body, giving it instructions to utilize former unknown systems that are ready to create spontaneous remission and beyond.

Remember my channelling given on the "youthing template" or the fact that you all have dormant "genesis cells" within your body (cells that have the full body blueprint and a source for very young stem cells)? These both were expositions of systems that are smart and ready to create new cells and healing in ways that will seem miraculous! But they need to be "instructed". 

Funding Will Trail for Awhile

How much money do you think is invested in the old ways? Right now, there is an industry of investment that is going to fail. So, do you think all this money will then be invested into the new methods? The answer is NO, not right away. Just like the North Korean leader's blindness, the old ways are just too addicting. "Keep it the same," they will yell. "The fall is temporary," they will say. "It always worked before, and it always will." Many investors will lose fortunes. The dysfunction is already being seen clearly. When you turn on your media at night, much of what you hear in the commercial breaks is how the chemistry didn't work! Now you get a chance to join others and sue the pharma companies for bad chemistry! You think something is beginning to break? It is. You see, they are already wallowing in something that's going to make them fail. They are advertising how broken the very system is. Both big pharma and their investors don't see the light coming yet, even though it is becoming dysfunctional before their eyes.  ..."

WHO launches genetic research register after designer-baby scandal

Yahoo – AFP, August 29, 2019

Chinese scientist He Jiankui, pictured in November 2018, altered the DNA of twin
girls in southern China by using molecular scissors -- ostensibly to prevent them
from contracting HIV (AFP Photo/Anthony WALLACE)

Geneva (AFP) - The WHO announced Thursday it will create a global registry to track research into human genetic manipulation, after a call to halt all work on germline genome editing -- used in China last year to genetically modify twin baby girls.

"New genome editing technologies hold great promise and hope for those who suffer from diseases we once thought untreatable," the World Health Organization's Director General for told the body's genome editing oversight committee meeting in Geneva.

"But some uses of these technologies also pose unique and unprecedented challenges -– ethical, social, regulatory and technical,” he added.

Chinese scientist He Jiankui's announcement last November that he had altered the DNA of twins girls in southern China by using molecular scissors -- ostensibly to prevent them from contracting HIV.

He was then fired from his university, put under police investigation and ordered to halt his work.

But his announcement provoked a global backlash from scientists saying the untested procedure was unethical and potentially dangerous and in December the WHO set up an expert committee to look into the matter.

About 30 nations currently have legislation directly or indirectly barring all clinical use of germline editing.

WHO chief Tedros Adhanom Ghebreyesus emphasised that countries should not allow any further work on human germline genome editing "until the technical and ethical implications have been properly considered", the WHO said in a statement.

Accepting the recommendation of its 18-member expert committee, WHO announced plans for an initial phase of the registry to include both germline and somatic clinical trials

Somatic mutations occur in a single body cell and cannot be inherited while germline mutations can be passed onto offspring.

Related Articles:

China eyes bans for rogue scientists after gene-editing uproar

Chinese hospital denies approving gene-edited babies experiment

Second HIV remission patient rekindles cure hope

Yahoo – AFP, Marlowe HOOD and Patrick GALEY, 5 March 2019

Ten years after the first confirmed case of an HIV-infected person being rid of the
deadly disease, a man known only as the 'London patient' has shown no sign of the
virus for nearly 19 months

For just the second time ever an HIV patient is in sustained remission from the virus in what was hailed by experts Tuesday as proof that the AIDS-causing condition could one day be curable.

Ten years almost to the day since the first confirmed case of an HIV-infected person being rid of the deadly disease, a man known only as the "London patient" has shown no sign of the virus for nearly 19 months, doctors reported in the journal Nature.

Both patients underwent bone marrow transplants to treat blood cancers, receiving stem cells from donors with a genetic mutation present in less than one percent of Europeans that prevents HIV from taking hold.

"It is a landmark. After 10 years of not being able to replicate (the first case), people were wondering if this was a fluke," said lead author Ravindra Gupta, a professor at the University of Cambridge.

"I think it is important to reaffirm that this is real and it can be done," Gupta told AFP.

The findings will be presented later Tuesday at a medical conference in Seattle, Washington.

Graphic on how HIV attacks white blood cells

Millions of people infected with HIV around the globe keep the disease in check with so-called antiretroviral therapy (ARV), but the treatment does not rid patients of the virus.

Close to 37 million people are living with HIV worldwide, but only 59 percent are receiving ARV. Nearly one million people die every year from HIV-related causes.

A new drug-resistant form of HIV is also a growing concern.

The first sustained remission survivor, announced in 2009 as "the Berlin patient" and later named as American Timothy Brown, was given two transplants and underwent total body irradiation to treat leukaemia -- a process that nearly killed him.

Gupta said that while a second successful transplant did not constitute a generalised cure, it showed that even milder forms of treatment can achieve full remission.

"There are a number of learning points here," he said. "Radiation has a lot of side-effects and leads to a delayed recovery of the bone marrow, so it's really good that we've shown you don't need radiation.

"The Berlin patient also had two rounds of chemotherapy because the first one didn't work. We've done ours just once, and it was also a milder form, which is important," he added.

Close to 37 million people are living with HIV worldwide, but only 59 percent 

are receiving treatment

'HIV is curable'

Both patients received stem cell transplants from donors carrying a genetic mutation that prevents expression of an HIV receptor, known as CCR5.

The London patient was diagnosed with HIV infection in 2003 and had been on antiretroviral therapy since 2012.

Later that year, he was diagnosed with advanced Hodgkin's Lymphoma, a deadly cancer.

He underwent a stem cell transplant in 2016 from a donor with two copies of a CCR5 gene variant, which is resistant to most HIV-1 virus strains.

"CCR5 is something essential for the virus to complete its life-cycle and we can't knock out many other things without causing harm to the patient," said Gupta.

"We know that CCR5 can be knocked out without any serious consequences because people are walking around without that gene."

Map showing the latest data on HIV infections for adults between 2010 to 2017

CCR5 was the target in the genome of the controversial gene-edited twins born last year in China, whose father is HIV-positive.

Experts cautiously welcomed Tuesday's announcement.

The International AIDS Society said in a statement Tuesday that results from the second patient "reaffirm our belief that there exists a proof of concept that HIV is curable".

Sharon Lewin, director of the Peter Doherty Institute for Infection and Immunity, told AFP that the second case showed a cure was "feasible".

"We can try to tease out which part of the transplant might have made a difference here, and allowed this man to stop his anti-viral drugs," she added.

New communities

After the bone marrow transplant, the London patient remained on ARV for 16 months, at which point treatment was stopped.

Regular testing has confirmed that the patient's viral load remained undetectable since then.

But scientists were keen to stress that the technique is likely only viable among a tiny percentage of sufferers.

The first sustained remission survivor, announced in 2009 as "the Berlin patient" and 

later named as American Timothy Brown, was given two transplants and underwent 

total body irradiation to treat leukaemia -- a process that nearly killed him

"Due to the rarity of suitable donors, this precise approach will not be available to all HIV patients," said Aine McKnight, professor of Viral Pathology at Queen Mary University, London.

"However, this work has the potential to stimulate research into more generally applicable therapies."

Gupta said he hoped to expand research on the stem-cell transplant technique to focus on communities in Africa, where the HIV-beating mutation does not naturally occur.

"Expanding remission to populations that are affected disproportionately is quite important," he told AFP.