Genetic Modification as Medicine

On ERV, Abbie Smith provides an update on a pioneering treatment for hemophilia that uses viruses to insert missing genes in a patient’s DNA. Hemophilia results from from the mutation or deletion of a gene that makes a blood clotting agent called Factor IX; without it, hemophiliacs are at risk for uncontrolled bleeding. While Factor IX can be delivered pharmaceutically, utilizing viruses to modify patients’ DNA yields long-term improvements in natural Factor IX production. Abbie writes, “the amount of therapeutic Factor IX these patients needed (on average) dropped from 2613 IU/kg to 206. The people who got the ‘high’ dose of virus dropped that down to 92 IU/kg. They went from 15-16 ‘bleeding episodes’ a year, to one.” They also saved $2.5 million.

Next, Abbie revisits research on treating HIV by removing CCR5 receptors that the virus uses to enter white blood cells. Much excitement was generated in 2008 when the “Berlin Patient” was declared to be functionally cured of HIV after receiving bone marrow from a donor with a mutation that preludes manufacture of the CCR5 protein. Now scientists are considering using gene therapy to disable CCR5 production in HIV patients, but there’s a catch: some HIV quasispecies utilize other receptor proteins, and even a small population of such viruses can take over when a patient is not producing CCR5. For this reason, Abbie writes that this therapy may hold more promise as a vaccine for HIV than as a cure.

Meanwhile, HIV itself has been genetically modified to help some sufferers of acute lymphoblastic leukemia by training cytotoxic T cells to target cancerous B cells. Abbie writes, “for all the time HIV has stolen from people, from families, its nice to see it giving some time back.”


Last Week on

Scientists use a ‘gene gun’ to insert a gene from a flowering plant called rockcress into the cells of wheat seeds. The genetically modified wheat became more resistant to a fungus called take-all, which in real life can cause “a 40-60% reduction in wheat yields.”

T-cells from six HIV+ patients were removed from their bodies, treated with a zinc-finger nuclease designed to snip a gene out of the cell’s DNA, and put back in the patients.  Removal of the gene mimics a naturally occurring mutation which confers resistance to the HIV virus.  But only 25% of the treated cells showed evidence of being successfully edited.

Researchers “use time-resolved X-ray microtomography to visualize the muscles and hinges in three-dimensions” of fly wings, modelling the complex physical processes that enable flies’ flight.

Even with the cost of building new energy storage infrastructure, wind energy will continue to offer a net gain of power.  Plus: wind produces enough surplus electricity to offer 72 hours of backup power (vs. 24 hours for solar panels).  Researchers say that the industry of onshore wind turbines can “double in size each year—and still maintain an energy surplus.”

Researchers cremated the remains of young piglets to investigate why there’s little evidence of high infant mortality in the archaeological record. To no avail.

Men in ‘traditional’ marriages (whose wives are not employed) are more likely to look negatively upon women in the workplace.

Regardless of the structural integrity of a shoulder (rotator cuff) repair, patients have improved function and reduced pain after surgery.

Stem cells are influenced by the rigidity of the substrate they grew up on: “spending 10 days on a particular bed leads to irreversible future differentiation of the stem cells into stiff-environment-loving bone or soft-loving fat cells.”  That could lead to considerable demand for a new scaffolding material “based on a biocompatible silk-alginate hydrogel” which can be made to varying standards of firmness.

By appearing to tap test subjects on the hand with a small hammer while playing the recorded sounds of a hammer tapping stone, researchers made people feel their hands were more stone-like (or numb).

A gene coincidentally named FAT10 ( for F Adjacent Transcript) actually “regulates lipid metabolism and longevity,” and model mice who lacked the gene were leaner, had a faster metabolism, and lived up to 20% longer.

Encapsulating immature pancreatic cells grown from human stem cells and implanting them under the skin of mice showed the cells could produce insulin whenever needed and reduce diabetic symptoms.

The CDC revised it autism prevalence rate upward again; in 2010 about 1 in 68 eight-year-olds had an autism spectrum disorder.

At age six, children award beneficial resources to members of their ‘in-group;’ at age eight they also assign harmful or negative resources to members of an ‘out-group.’

People with OCD were less likely than controls to believe they could influence a light bulb by pressing a space bar whenever they want. The light bulb blinked randomly on and off.

A virus affecting crickets not only sterilizes them, but makes them more eager to initiate courtship. Males perhaps uninhibited by the virus would start playing a courtship song for a female much sooner than their healthy peers.  Intimacy may be the virus’s way of spreading.

Within the ultrapure water purification system of a nuclear reactor, scientists found oligotrophic bacteria, including new species, growing in biofilms “visible to the naked eye” on ceramic filter surfaces.

Discrediting Anti-GMO Propaganda

Not too long ago, when the media became excited about a study saying genetically modified corn causes tumor growth in rats, ScienceBloggers were quick to point out that the study featured some of the worst science ever.  Now the paper has been officially retracted by its publisher, but to what end?  On Pharyngula, PZ Myers speculates that the study authors avoided statistical analysis of their small, cancer-prone rat packs precisely because there was no statistically significant effect of being force-fed GMO corn.  PZ also says “journalists who got the paper in advance had to sign confidentiality agreements that prohibited them from consulting with experts,” leading to uncritical promulgation of a story well-suited to stoke fear and anti-GMO politics.  Even the rats in the study were treated unethically, forced to live out their tumor-ridden days for the sake of sensational photographs.  On Respectful Insolence, Orac wonders if the paper’s retraction will only fuel the convictions of activists pre-disposed to conspiratorial thinking.  And on ERV, Abbie Smith offers a book of actual science on GMOs compiled by actual scientists and farmers, for free.

Posted to the homepage on December 2, 2013.