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Thread: Sickle Cell & Other Blood-related Disorders

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    Lightbulb Sickle Cell & Other Blood-related Disorders

    Human Trials Planned to use CRISPR to Correct Sickle Cell...

    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    With the study, and unpublished findings from his lab, Porteus believes his team has amassed enough proof to start planning the first human clinical trial using the powerful CRISPR-Cas9 gene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.


    Red blood cells in a sickle cell patient, after a bone marrow transplant at the National Institutes of Health Clinical Center in Bethesda, Md. Medical Association.

    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.

    http://www.voanews.com/a/reu-stanfor...d/3586115.html

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    CRISPR-Cas9 may be used to remove genes that cause inherited diseases...

    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.


    A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute to Reuters on May 15, 2012. A group of 25 scientists June 2, 2016, proposed an ambitious project to create a synthetic human genome, or genetic blueprint, in an endeavor that is bound to raise concerns over the extent to which human life can or should be engineered.

    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.

    http://www.voanews.com/a/scientists-...s/3724179.html

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    Big step forward...

    Human blood stem cells grown in the lab for the first time
    17 May 2017 - The stem cells that produce our blood have been created in the lab for the first time. These could one day be used to treat people who have blood diseases and leukaemia with their own cells, rather than bone marrow transplants from a donor. They could also be used to create blood for transfusions.
    “This is a very big deal,” says Carolina Guibentif at the University of Cambridge, who was not involved in the research. “If you can develop [these cells] in the lab in a safe way and in high enough numbers, you wouldn’t be dependent on donors.” In a healthy adult, blood stem cells are found in bone marrow, where they replenish the supply of red and white blood cells and platelets. “They are sort of master cells,” says George Daley at Harvard Medical School.

    When these cells don’t work properly, they fail to maintain an adequate supply of blood cells. As a result, not enough oxygen reaches the body’s tissues. This can cause serious disease if organs such as the heart are affected. Blood stem cells can also be wiped out by chemotherapy for leukaemia and other cancers. People with these disorders tend to be treated with bone marrow – complete with blood stem cells – from a healthy donor. The difficulty is finding a match. There is a one in four chance of achieving this from a healthy sibling, but the odds are slashed to one in a million if a stranger needs to be found, says Daley.

    Making cells

    In an attempt to create blood stem cells in the lab, Daley and his colleagues started with human pluripotent stem cells – which have the potential to form almost any other type of body cell. The team then searched for chemicals that might encourage these to become blood stem cells. After studying the genes involved in blood production, the researchers identified proteins that control these genes and applied them to their stem cells.


    Potential for a new supply line

    They tested many combinations of the proteins, and found five that worked together to encourage their stem cells to become blood stem cells. When they put these into mice, they went on to produce new red and white blood cells and platelets. “It’s very cool,” says Daley. “We’re very excited about the results.” A separate team has achieved the same feat with stem cells taken from adult mice. Raphael Lis at Weill Cornell Medical College in New York and his colleagues started with cells taken from the walls of the animals’ lungs, based on the idea that similar cells in an embryo eventually form the body’s first blood stem cells. The team identified a set of four factors that could encourage these lung stem cells to make them.

    Big step forward

    Both sets of results represent a “breakthrough”, says Guibentif. “This is something people have been trying to achieve for a long time,” she says. By working with adult mouse epithelial cells, Lis and his team show that the feat could potentially be achieved with cells taken from an adult person. Daley’s team used human stem cells that could in theory be made from skin cells, bolstering the prospect that lab-made human blood could be next. The lab-made stem cells are not quite ready to be used in people just yet, says Daley. Although all of his mice were healthy throughout the experiments, there is a risk that the cells could mutate and cause cancer. And the cells are not quite as efficient at making blood as those found in the body.

    But once Daley and his team have honed their procedure, they might be able to make platelets and red blood cells for hospital use. These cell types don’t have a nucleus, so are unable to divide and potentially cause cancer. He hopes this procedure could be used within the next couple of years. Eventually, Daley hopes his cells could be used to create whole blood suitable for transfusions. Not only would such a supply be more reliable than that from donors, but it would also be free of disease. “When new pathogens like Zika pop up, you have to make sure that blood is safe,” says Daley. “We’d be able to have more quality control.”

    https://www.newscientist.com/article...he-first-time/

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