A startup called Strata Oncology says it plans to give away advanced genetic tests to 100,000 patients struggling with cancer. But there's a profit motive: it hopes to identify patients with specific rare DNA errors and steer them to drug companies.
Strata, which was founded last year and has raised $12 million from investors, says it is set up to run 50,000 next-generation sequencing tests a year. Such tests probe the DNA of tumor tissue, searching for mutations in hundreds of genes at once, hoping to surface clues about what drug a patient should be taking.
A federal safety board next week will consider the first human use of the gene-editing technology CRISPR, according to the National Institutes of Health.
The proposed treatment is an immune therapy in which a patient’s own blood cells will be removed and genetically altered using the technology, a type of molecular scissors able to precisely cut DNA.
The cancer treatment, in development by the University of Pennsylvania, is designed to target myeloma, melanoma, and sarcomas, according to the NIH.
The aim of nanoparticle self-assembly research has been to get the particles to organize themselves into structures arrangment is largely controlled by us. However, the level of control we have sought has sometimes left a little bit to be desired.
Earlier this spring, Bill Maurits sat in a waiting room in Philadelphia ready to have a trillion viruses dripped into his body through an I.V. “I was like, ‘Yeah, let’s go. I can’t wait,’” he says.
Maurits has hemophilia B, which means his body doesn’t produce factor IX, a protein that clots blood. He’ll get a gusher if he gets cut, and his joints are like knotted branches from all the bruises. Since he was 10, he’s depended on injections of “ridiculously expensive” replacement protein. Lately, his left ankle has been killing him.
A treatment now pending approval in Europe will be the first commercial gene therapy to provide an outright cure for a deadly disease.
The treatment is a landmark for gene-replacement technology, an idea that’s struggled for three decades to prove itself safe and practical.
Called Strimvelis, and owned by drug giant GlaxoSmithKline, the treatment is for severe combined immune deficiency, a rare disease that leaves newborns with almost no defense against viruses, bacteria, or fungi and is sometimes called “bubble boy” disease after an American child whose short life inside a protective plastic shield was described in a 1976 movie.
A proposal by a group of scientists and businesspeople to synthesize a human genome from scratch is attracting sharp criticism for dodging the big ethical questions such a step raises.
The proposal, described today in a two-and-a-half-page letter being published in Science, is to string together synthetically made DNA and shape from it a human genome able to power a cell in a dish, according to lead authors Jef Boeke of New York University’s Langone Medical Center and biotechnologist George Church of Harvard Medical School.
Yes, it’s true that a group of leading geneticists is calling for the construction of a synthetic human genome. That means they want to take 3 billion chemical building blocks and assemble them into one complete package of DNA, encoding all the body parts and life processes that make up a functional human being.
But the organizers want to make one thing very clear: “We’re not planning to make synthetic people,” says a somewhat exasperated Jef Boeke, one of the champions of this proposal. “We never were.”
When Sean Harper took over as Amgen’s head of research in 2012, one of his first moves was to get the biotech giant to pay $415 million to buy DeCode Genetics, a struggling company in Iceland well-known for the huge DNA database it had built.
Today that bet paid off as scientists at DeCode describe how they found a specific DNA defect that lowers the chance of having a heart attack by 35 percent, which they call the largest such effect ever found.
DeCode’s DNA detective work is “outstanding,” says Sekar Kathiresan, a scientist at the MIT/Harvard Broad Institute in Cambridge, Massachusetts. “This work immediately suggests that a medicine which mimics the protective mutation should reduce risk for heart attack.”
A group of biotech veterans have debuted today a new company, Homology Medicine, with a bold claim that their underlying science is a better version of the gene editing methods, such as CRISPR-Cas9, that have captured the attention of patients, doctors, and scientists looking to treat desperate diseases.
The claim is, for now, untested, as none of the work has been published. One of the company’s backers says Homology will publish some of its findings soon.
Nonie Hickle’s hair is coal-dark. Her husband, Vincent, marvels at it. He points to it and says, “No gray!” Nonie’s a little amazed too; she thinks it’s kind of spooky. After all, she’s 91 years old.
Statistically speaking, Hickle, who lives in San Diego’s Hillcrest neighborhood, should be sick. She ought to have cardiovascular disease, cancer, or heart failure. Yet she doesn’t have any of those things. True, her hearing has been going bad over the past several years. And she has a touch, just a touch, of high blood pressure. But if you were to look at this 4'11" Korean-American nonagenarian, you wouldn’t peg her at a day over 70.
