Lifeboat Foundation

A remarkable new study has successfully used the CRISPR-Cas9 gene editing technique to edit a specific gene in mice engineered to have fragile X syndrome (FXS), a single-gene disorder often related to autism. The single gene edit in the live mice resulted in significant improvements in repetitive and obsessive behaviors, making this the first time gene editing has been used to effectively target behavioral symptoms related to autism spectrum disorder (ASD).

FXS is a genetic disorder associated with intellectual disability, seizures and exaggerated repetitive behavior. Previous studies have shown that the repetitive behaviors associated with FXS are related to a specific excitatory receptor in the brain that, when dysregulated, causes exaggerated signaling between cells.

The CRISPR technique homes in on the gene that controls that excitatory receptor, the metabotropic glutamate receptor 5 (mGluR5), and essentially disables it, dampening the excessive signaling the corresponds with repetitive behaviors. In mice treated with the new system, obsessive digging behavior was reduced by 30 percent and repetitive leaping actions dropped by 70 percent.

Chemical innovation plays a key role in developing cutting-edge technologies for the military. Research chemists design and synthesize new molecules that could enable a slew of next-generation military products, such as novel propellants for spacecraft engines; new pharmaceuticals and medicines for troops in the field; lighter and longer-lasting batteries and fuel cells; advanced adhesives, coatings and paints; and less expensive explosives that are safer to handle. The problem, however, is that existing molecule design and production methods rely primarily on experts’ intuition in a laborious, trial-and-error research process.

DARPA’s Make-It program, currently in year three of a four-year effort, is developing software tools based on machine learning and expert-encoded rules to recommend synthetic routes (i.e., the “recipe” to make a particular molecule) optimized for factors such as cost, time, safety, or waste reduction. The program seeks to free chemists so that they may focus their energy on chemical innovation, rather than testing various molecular synthesis pathways. The program also is developing automated devices that uniformly and reproducibly create the desired chemical based on the software-generated recipe – this one-device, many-molecules concept is a departure from the traditional dedicated reactors in chemical production. Make-It research teams have recently demonstrated significant progress toward fully automated rapid molecule production, which could speed the pace of chemical discovery for a range of defense products and applications.

“A seasoned research chemist may spend dozens of hours designing synthetic routes to a new molecule and months implementing and optimizing the synthesis in a lab,” said Anne Fischer, program manager in DARPA’s Defense Sciences Office. “Make-It is not only freeing chemists to expend brain power in other areas such as molecular discovery and innovation, it is opening chemical synthesis and discovery to a much broader community of scientific researchers who will benefit from faster development of new molecules.”

Today, we have part one of a two-part interview with Dr. Michael Fossel, the driving force behind Telocyte, a new company focused on telomerase therapy for various diseases, and a strong advocate of telomerase therapy to treat human disease over the past three decades.

I interviewed Dr. Fossel as an individual thought leader in this field and not in his role representing Telocyte, so the opinions stated here are purely his own.

Born in 1950, Michael Fossel grew up in New York and lived in London, Palo Alto, San Francisco, Portland, and Denver. He graduated cum laude from Phillips Exeter Academy, received a joint B.A. and M.A. in psychology in four years from Wesleyan University in Connecticut, and, after completing a Ph.D. in neurobiology at Stanford University in 1978, went on to finish his M.D. at Stanford Medical School in two and a half years. He was awarded a National Science Foundation Fellowship and taught at Stanford University, where he began studying aging with an emphasis on premature aging syndromes. Dr. Fossel was a Clinical Professor of Medicine at Michigan State University for almost three decades and taught the Biology of Aging at Grand Valley State University.

At MBC Biolabs, an incubator for biotech startups in San Francisco’s Dogpatch neighborhood, a team of scientists and interns working for the small startup Prellis Biologics have just taken a big step on the path toward developing viable 3D-printed organs for humans.

The company, which was founded in 2016 by research scientists Melanie Matheu and Noelle Mullin, staked its future (and a small $3 million investment) on a new technology to manufacture capillaries, the one-cell-thick blood vessels that are the pathways which oxygen and nutrients move through to nourish tissues in the body.

Continue reading “Implantable 3D-printed organs could be coming sooner than you think” »

Research team successfully administers insulin to rats in capsule form, raising hopes that a version for humans could be developed.

Nicola Davis

Engineered strains of E. coli and other microbes are being tested in humans to combat a slew of illnesses.

TRX-1 inhibitor TXNIP might be implicated in increased oxidative stress as we age.

According to scientists at the German Cancer Research Center (Deutsches Krebsforschungszentrum, or DKFZ), the enzyme TXNIP, which inhibits the enzyme TRX-1, might be a regulator of aging and might be a viable candidate for future interventions against age-related diseases [1].

Study summary

Continue reading “Antioxidant inhibitor might be a regulator of aging” »

An international team of researchers have developed a low-cost sensor made from semiconducting plastic that can be used to diagnose or monitor a wide range of health conditions, such as surgical complications or neurodegenerative diseases.

The sensor can measure the amount of critical metabolites, such as lactate or glucose, that are present in sweat, tears, saliva or blood, and, when incorporated into a diagnostic device, could allow health conditions to be monitored quickly, cheaply and accurately. The new device has a far simpler design than existing sensors, and opens up a wide range of new possibilities for health monitoring down to the cellular level. The results are reported in the journal Science Advances.

The device was developed by a team led by the University of Cambridge and King Abdullah University of Science and Technology (KAUST) in Saudi Arabia. Semiconducting plastics such as those used in the current work are being developed for use in solar cells and flexible electronics, but have not yet seen widespread use in biological applications.

Continue reading “Low-cost plastic sensors could monitor a range of health conditions” »

This is the Freethink Dispatch, our rundown of the stories that mattered from the frontiers of a changing world. This week, engineers created brain-surgery robots that can work inside an MRI, scientists found a way to use coffee to treat diabetes, and a startup is making fresh produce that lasts twice as long. All that and more, plus a new episode of Freethink’s original hit series Superhuman about how doctors are reprogramming the immune system to kill untreatable cancers.