Gene editing

[waltky]

Mebbe gene editing is what caused zika?...

Scientists Say Gene Editing Should Stay in Labs
June 09, 2016 | WASHINGTON — Much as they did at the onset of the Atomic Age, scientists are now grappling with the ethics of a technological advancement — in this case, our ability to manipulate the genetic code.

And they warn that civilization is rapidly approaching the point where it will have to decide what to do with its newly acquired power to change the genetic structure of living things. The newest conversation has to do with an enhanced inheritance system known as the “gene drive,” a self-replicating bit of genetic code that scientists could use to pass on created genetic traits to the offspring of a living thing in the wild. We modify the genetics of things we control all the time, Mendel's plants for example, and we cross-breed domesticated animals and agricultural products to make them more productive, or adaptive to harsh climates.

Professor Wendy Harwood poses for a photograph in a plant breeding incubator room with barley plants that have undergone gene editing at the John Innes Center in Norwich, Britain

But inserting a modified bit of DNA into a gene drive makes it possible to pass along genetic traits to plants and animals in the wild, even traits that reduce their fitness for survival. Conceivably, scientists could use a gene drive to wipe out the Aedes aegypti mosquitoes that carry the Zika virus, or invasive species that are destroying native plants and animals.

Power, peril of gene drive

In a report published Wednesday, the U.S. National Academies of Sciences, Engineering and Medicine endorsed research on the technology but also warned that nobody knows whether the release of altered organisms into the wild would cause some unwanted consequences. The NAS committee, made up of 16 biologists, ethicists and policy makers, says that in spite of clear potential benefits of the new technology, there is not enough evidence to support any release of modified organisms into the environment. For understandable reasons, many people would support disabling organisms such as mosquitoes and some rodents from carrying the diseases.

Aedes aegypti mosquitoes are seen in a mosquito cage at a laboratory in Cucuta, Colombia

But scientists say before doing that we need to fully understand the scientific, ethical, regulatory and social consequences of such a move. Scientists say, unlike pesticides or genetically modified organisms, the “gene drive” is spread through sexual reproduction and will keep spreading as long as the target organism keeps reproducing. It cannot be localized within a farm or within a country’s borders. In addition, if a modified organism mates with another species, the gene drive could spread to the non-target species, perhaps driving it into extinction.

http://www.voanews.com/content/scien...s/3368789.html

[Peter1469]

Here is the article from Naturenews.


Others were saying this years ago.

[waltky]

Mebbe dey'll be able to filter out genetic disorders?...

Scientists in US Successfully Edit Human Embryo's Genes
July 27, 2017 - Scientists at the Oregon Health and Science University say they have successfully edited genes of human embryos in the first such attempt in the United States.

Previously, similar experiments have been reported only by scientists in China. Engineering human genes in the embryo stage opens up the possibility of correcting their defective parts that cause inherited diseases. The new trait is passed on to subsequent generations. But the practice is controversial, since many fear it could be used for unethical purposes such as creating "designer babies" with specific enhanced abilities or traits.

A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute. For the first time, U.S. scientists have successfully edited genes of human embryos.

Oregon scientists led by Kazakhstan-born Shoukhrat Mitalipov successfully repeated the experiment on scores of embryos created with sperm donated for scientific purposes by men with inherited disease mutations. The editing was done very close to the moment of fertilization of the egg in order to make sure the changes would be repeated in all subsequent cells of the embryo.

Scientists have been experimenting with gene editing for a long time, but the availability of the technique called CRISPR rapidly advanced the precision, flexibility and efficiency of cutting and replacing parts of the molecule chains that comprise genes. Citing ethical concerns, the U.S. Congress made it illegal to turn genetically-edited embryos into babies. Many other countries do not have such regulations.

https://www.voanews.com/a/us-scienti...s/3961745.html

See also:

Scientists Soften on DNA Editing of Human Eggs, Sperm, Embryos: Report
February 14, 2017 - Although not ready yet, powerful gene editing tools may one day be used on human embryos, eggs and sperm to remove genes that cause inherited diseases, according to a report by U.S. scientists and ethicists released on Tuesday.

The report from the National Academy of Sciences (NAS) and the National Academy of Medicine said scientific advances make gene editing in human reproductive cells "a realistic possibility that deserves serious consideration.” The statement signals a softening in approach over the use of the technology known as CRISPR-Cas9, which has opened up new frontiers in genetic medicine because of its ability to modify genes quickly and efficiently. In December 2015, scientists and ethicists at an international meeting held at the NAS in Washington said it would be "irresponsible" to use gene editing technology in human embryos for therapeutic purposes, such as to correct genetic diseases, until safety and efficacy issues are resolved.

The latest NAS report now says clinical trials for genome editing of the human germline could be permitted, "but only for serious conditions under stringent oversight." CRISPR-Cas9 works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA. Genome editing is already being planned for use in clinical trials of people to correct diseases caused by a single gene mutation, such as sickle cell disease. But these therapies affect only the patient.

The concern is over the use of the technology in human reproductive cells or early embryos because the changes would be passed along to offspring. Research using the powerful technique is plowing ahead even as researchers from the University of California and the Broad Institute battle for control over the CRISPR patent. Although gene editing of human reproductive cells to correct inherited diseases "must be approached with caution, caution does not mean prohibition," the committee said in a statement.

https://www.voanews.com/a/scientists...s/3724179.html

Related:

Stanford Uses CRISPR to Correct Sickle Cell, Human Trials Planned
November 08, 2016 — Scientists at Stanford University School of Medicine have used the CRISPR gene editing tool to repair the gene that causes sickle cell disease in stem cells from diseased patients, paving the way for a potential cure for the disease, which affects up to 5 million people globally.

"What we've finally shown is that we can do it. It's not just on the chalkboard," said Dr. Matthew Porteus, senior author of the study published in the journal Nature. With the study, and unpublished findings from his lab, Porteus believes his team has amassed enough proof to start planning the first human clinical trial using the powerful CRISPR-Cas9 gene editing system to correct the genetic mutation that causes sickle cell disease. "We think we have a complete data set to present to the FDA (Food and Drug Administration) to say we've done all pre-clinical experiments to show this is ready for a clinical trial," Porteus told Reuters by phone. CRISPR-Cas9 has quickly become the preferred method of gene editing in research labs because of its ease of use compared with older techniques. CRISPR works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA.

Research using the powerful technique is plowing ahead even as researchers from the University of California and the Broad Institute battle for control over the CRISPR patent. Oral arguments in the case are expected on Dec. 6 at the U.S. Patent and Trademark Office in Alexandria, Va. In sickle cell disease, the body makes mutant, sickle-shaped hemoglobin, the protein in red blood cells that carries oxygen to the body's tissues. It is caused by a single mutation in a gene that makes a hemoglobin protein. In a study published last month in Science Translational Medicine, a team from the University of California, Berkeley, and colleagues used the CRISPR gene editing tool to snip out the diseased gene and deliver a new stretch of DNA to correct the mutation in human stem cells. In that study, some 25 percent of blood-forming cells were corrected.

In the Stanford study, Porteus and colleagues took a different approach. They used CRISPR to snip the gene, but they used a harmless virus to introduce the repair mechanism into cells. After a series of tests in healthy cells, the team tested the gene editing system in blood-forming cells from four patients with sickle cell disease. They showed they could correct the mutation in 30 to 50 percent of these diseased cells. Sixteen weeks after they injected the cells into young mice, the team found the cells were still thriving in the bone marrow. Porteus said the findings were very encouraging because prior studies have shown that if you can correct mutations in 10 percent of cells, that should create enough to cure the disease. Stanford is now scaling up its laboratory processes to support human trials.

The process will involve using chemotherapy to wipe out a patient's blood system but not their immune system, as is done in a stem cell transplant. Then, the team would inject the patient's own corrected stem cells, which the researchers hope would engraft into the bone marrow and produce healthy blood cells. Porteus has equity interest in CRISPR Therapeutics of Cambridge, Massachusetts, but he said the sickle cell work has been independent of it. The university has built a cell manufacturing plant for this purpose. "We hope to develop the entire process here at Stanford," he said. Porteus said the team plans to make an initial submission to the FDA in the next few months to map out the clinical trial, and hopes to treat the first patient in 2018.

https://www.voanews.com/a/reu-stanfo...d/3586115.html

[waltky]

Cut-and-Paste Gene Editing...

Like a Cut-and-Paste Tool, Gene Editing Transforms Research
August 02, 2017 | WASHINGTON — Gene editing is getting fresh attention thanks to a successful lab experiment with human embryos. But for all the angst over possibly altering reproduction years from now, this technology already is used by scientists every day in fields ranging from agriculture to drug development.

New gene editing tools let scientists alter the DNA of living cells - from plants, animals, even humans - more precisely than ever before. Think of it as a biological cut-and-paste program. A look at the science.


What is gene editing?


While scientists have long been able to find defective genes, fixing them has been so cumbersome that it's slowed development of genetic therapies. There are several gene editing methods, but a tool called CRISPR-Cas9 has sparked a boom in research as laboratories worldwide adopted it over the past five years because it's faster, cheaper, simple to use with minimal training and allows manipulation of multiple genes at the same time.


How it works


Pieces of RNA are engineered to be a guide that homes in on the targeted stretch of genetic material. The Cas9 is an enzyme that acts like molecular scissors to snip that spot. That allows scientists to delete, repair, or replace a particular gene.


Medical research


The fresh attention comes from research involving human embryos. In laboratory experiments, a team lead by Oregon researchers used CRISPR to successfully repair a heart-damaging gene in human embryos, marking a step toward one day being able to prevent inherited diseases from being passed on to the next generation. But there's wide agreement that more research is needed before ever testing the technique in pregnancy.

Professor Wendy Harwood poses for a photograph in a plant breeding incubator room with barley plants that have undergone gene editing at the John Innes Centre in Norwich, Britain.

The biggest everyday use of CRISPR so far is to engineer animals with human-like disorders for basic research, such as learning how genes cause disease or influence development and what therapies might help. But promising research, in labs and animals so far, also suggests gene editing might lead to treatments for such diseases as sickle cell, cancer, maybe Huntington's _ by altering cells and returning them to the body. Another project aims to one day grow transplantable human organs inside pigs.


The biggest hurdle

See also:


US Scientists Able to Alter Genes of Human Embryos
August 02, 2017 - U.S. scientists have succeeded in altering the genes of a human embryo to correct a disease-causing mutation, making it possible to prevent the defect from being passed on to future generations.

The milestone, published this week in the journal Nature, was confirmed last week by Oregon Health and Science University (OHSU), which collaborated with the Salk Institute and Korea's Institute for Basic Science to use a technique known as CRISPR-Cas9 to correct a genetic mutation for a heart condition. Until now, published studies using the technique had been done in China with mixed results.


CRISPR-Cas9 works as a type of molecular scissors that can selectively trim away unwanted parts of the genome, and replace it with new stretches of DNA. "We have demonstrated the possibility to correct mutations in a human embryo in a safe way and with a certain degree of efficiency," said Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory and a co-author of the study.

Juan Carlos Izpisua Belmonte, professor at Salk Institute's Gene Expression Laboratory and Jun Wu, Salk staff scientists

To increase the success rate, his team introduced the genome editing components along with sperm from a male with the targeted gene defect during the in vitro fertilization process. They found that the embryo used the available healthy copy of the gene to repair the mutated part. The Salk/OHSU team also found that its gene correction did not cause any detectable mutations in other parts of the genome - a major concern for gene editing.


Still, the technology was not 100 percent successful - it increased the number of repaired embryos from 50 percent, which would have occurred naturally, to 74 percent. The embryos, tested in the laboratory, were allowed to develop for only a few days. "There is still much to be done to establish the safety of the methods, therefore they should not be adopted clinically," Robin Lovell-Badge, a professor at London's Francis Crick Institute who was not involved in the study, said in a statement.


'Utmost Caution'

[gamewell45]

Mebbe gene editing is what caused zika?...

Scientists Say Gene Editing Should Stay in Labs
June 09, 2016 | WASHINGTON — Much as they did at the onset of the Atomic Age, scientists are now grappling with the ethics of a technological advancement — in this case, our ability to manipulate the genetic code.

Interesting article; thanks for posting it.

[Bo-4]

More recent story about that here. Lot of crazy implications:

https://www.nytimes.com/2017/08/02/s...n-embryos.html

[waltky]

Studies could lead to new treatments for inherited diseases...

'Incredible' editing of life's building blocks
Wed, 25 Oct 2017 - The studies could lead to new treatments for inherited diseases.

Scientists have demonstrated an "incredibly powerful" ability to manipulate the building blocks of life in two separate studies. One altered the atoms in DNA to rewrite the human genetic code and the instructions for life. The other edited RNA, which is a chemical cousin of DNA and unlocks the information in the genetic code.

The studies - which could eventually treat diseases - have been described as clever, important and exciting. Cystic fibrosis, inherited blindness and other diseases caused by a single typo in the genetic code could ultimately be prevented or treated with such approaches. Both studies were performed at the Broad Institute of MIT and Harvard.

Base editing

The first, published in the journal Nature, developed tools called base editors. DNA is built out of the four bases: adenine (A), cytosine (C), guanine (G) and thymine (T). If a single one of them is in the wrong place, it can cause disease. Base editors alter the atomic structure of one base to convert it into another. Researchers can now manipulate the four bases. And the team used base editing to correct an inherited disease that leads to dangerously high levels of iron in the blood.

Prof David Liu of the Broad Institute said: "We are hard at work trying to translate base editing technology into human therapeutics." However, he admits there are still issues around safety and implementation: "Having a machine that can make the change you want to make is only the start. You still need to do all this other work, but having the machine really helps."

RNA

[waltky]

US Scientists Try 1st Gene Editing in the Body...

AP Exclusive: US Scientists Try 1st Gene Editing in the Body
November 15, 2017 — Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease.

The experiment was done Monday in California on 44-year-old Brian Madeux. Through an IV, he received billions of copies of a corrective gene and a genetic tool to cut his DNA in a precise spot. "It's kind of humbling" to be the first to test this, said Madeux, who has a metabolic disease called Hunter syndrome. "I'm willing to take that risk. Hopefully it will help me and other people." Signs of whether it's working may come in a month; tests will show for sure in three months.

If it's successful, it could give a major boost to the fledgling field of gene therapy. Scientists have edited people's genes before, altering cells in the lab that are then returned to patients. There also are gene therapies that don't involve editing DNA. But these methods can only be used for a few types of diseases. Some give results that may not last. Some others supply a new gene like a spare part, but can't control where it inserts in the DNA, possibly causing a new problem like cancer.

Brian Madeux sits with his girlfriend Marcie Humphrey while waiting to receive the first human gene editing therapy at the UCSF Benioff Children's Hospital in Oakland, Calif.

This time, the gene tinkering is happening in a precise way inside the body. It's like sending a mini surgeon along to place the new gene in exactly the right location. "We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending," said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. "It becomes part of your DNA and is there for the rest of your life."

That also means there's no going back, no way to erase any mistakes the editing might cause. "You're really toying with Mother Nature" and the risks can't be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.

Protections are in place to help ensure safety, and animal tests were very encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies. He said gene editing's promise is too great to ignore. "So far there's been no evidence that this is going to be dangerous," he said. "Now is not the time to get scared."

Woe from head to toe

See also:

FDA Approves First Digital Ingestion Tracking System Med
November 14, 2017 | WASHINGTON — The Food and Drug Administration has approved the first drug in the United States with a digital ingestion tracking system, in an unprecedented move to ensure that patients with mental illness take the medicine prescribed for them.

The drug Abilify MyCite was developed by Otsuka Pharmaceutical Co., Ltd. The drug Abilify was first approved by the FDA in 2002 to treat schizophrenia, and the ingestible sensor, made by Proteus Digital health, was approved for marketing in 2012. The FDA said in a statement Monday that the digitally enhanced medication “works by sending a message from the pill's sensor to a wearable patch.” “Being able to track ingestion of medications prescribed for illness may be useful for some patients,” said Dr. Mitchell Mathis, director of the division of Psychiatry Products in the FDA's Center for Drug Evaluation and Research. “The FDA supports the development and use of new technology in prescription drugs and is committed to working with companies to understand how this technology might benefit patients and prescribers.”

Green-lighting the new medication, however, came with some caveats. Among them, the FDA said it was important to note that Abilify MyCite's labeling asserts “the ability of the product to improve patient compliance with their treatment regimen has not been shown.” “Abilify MyCite should not be used to track drug ingestion in ‘real-time’ or during an emergency,” the statement said, “because detection may be delayed or may not occur.” In a portion of the statement that appeared to address privacy concerns, the FDA said the wearable patch that comes with the medication “transmits the information to a mobile application so that patients can track the ingestion of the medication on their smart phone. Patients can also permit their caregivers and physician to access the information through a web-based portal.”

In a statement issued last May at the time the FDA accepted submission of product for review, Otsuka Pharmaceutical, Ltd. of Toyko and Proteus Digital, of Redwood City, California, said that “with the patient's consent, this information could be shared with their health care professional team and selected family and friends, with the goal of allowing physicians to be more informed in making treatment decisions that are specific to the patient's needs.” The companies said the Proteus Ingestible sensor “activates when it reaches stomach fluids and communicates with the patch.” The FDA said the product is designed for the treatment of schizophrenia, acute treatment of manic and mixed episodes associated with a bipolar disorder and for use as an add-on treatment for depression in adults.”

https://www.voanews.com/a/fda-digita...g/4114427.html