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genetic engineering

Creative Minds: Building a CRISPR Gene Drive Against Malaria

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Valentino Gantz

Valentino Gantz/Credit: Erik Jepsen

Researchers have used Drosophila melanogaster, the common fruit fly that sometimes hovers around kitchens, to make seminal discoveries involving genetics, the nervous system, and behavior, just to name a few. Could a new life-saving approach to prevent malaria be next? Valentino Gantz, a researcher at the University of California, San Diego, is on a path to answer that question.

Gantz has received a 2016 NIH Director’s Early Independence Award to use Drosophila to hone a new bioengineered tool that acts as a so-called “gene drive,” which spreads a new genetically encoded trait through a population much faster than would otherwise be possible. The lessons learned while working with flies will ultimately be applied to developing a more foolproof system for use in mosquitoes with the hope of stopping the transmission of malaria and potentially other serious mosquito-borne diseases.


Cool Videos: Insulin from Bacteria to You

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If you have a smartphone, you’ve probably used it to record a video or two. But could you use it to produce a video that explains a complex scientific topic in 2 minutes or less? That was the challenge posed by the RCSB Protein Data Bank last spring to high school students across the nation. And the winning result is the video that you see above!

This year’s contest, which asked students to provide a molecular view of diabetes treatment and management, attracted 53 submissions from schools from coast to coast. The winning team—Andrew Ma, George Song, and Anirudh Srikanth—created their video as their final project for their advanced placement (AP) biology class at West Windsor-Plainsboro High School South, Princeton Junction, NJ.


Creative Minds: The Human Gut Microbiome’s Top 100 Hits

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Michael Fishbach

Michael Fishbach

Microbes that live in dirt often engage in their own deadly turf wars, producing a toxic mix of chemical compounds (also called “small molecules”) that can be a source of new antibiotics. When he started out in science more than a decade ago, Michael Fischbach studied these soil-dwelling microbes to look for genes involved in making these compounds.

Eventually, Fischbach, who is now at the University of California, San Francisco, came to a career-altering realization: maybe he didn’t need to dig in dirt! He hypothesized an even better way to improve human health might be found in the genes of the trillions of microorganisms that dwell in and on our bodies, known collectively as the human microbiome.


Sickle Cell Disease: Gene-Editing Tools Point to Possible Ultimate Cure

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Sickled red blood cell

Caption: An electron micrograph showing two red blood cells deformed by crystalline hemoglobin into different “sickle” shapes characteristic of people with sickle cell disease.
Credit: Frans Kuypers: RBClab.com, UCSF Benioff Children’s Hospital Oakland

Scientists first described the sickle-shaped red blood cells that give sickle cell disease its name more than a century ago. By the 1950s, the precise molecular and genetic underpinnings of this painful and debilitating condition had become clear, making sickle cell the first “molecular disease” ever characterized. The cause is a single letter “typo” in the gene encoding oxygen-carrying hemoglobin. Red blood cells containing the defective hemoglobin become stiff, deformed, and prone to clumping. Individuals carrying one copy of the sickle mutation have sickle trait, and are generally fine. Those with two copies have sickle cell disease and face major medical challenges. Yet, despite all this progress in scientific understanding, nearly 70 years later, we still have no safe and reliable means for a cure.

Recent advances in CRISPR/Cas9 gene-editing tools, which the blog has highlighted in the past, have renewed hope that it might be possible to cure sickle cell disease by correcting DNA typos in just the right set of cells. Now, in a study published in Science Translational Medicine, an NIH-funded research team has taken an encouraging step toward this goal [1]. For the first time, the scientists showed that it’s possible to correct the hemoglobin mutation in blood-forming human stem cells, taken directly from donors, at a frequency that might be sufficient to help patients. In addition, their gene-edited human stem cells persisted for 16 weeks when transplanted into mice, suggesting that the treatment might also be long lasting or possibly even curative.


Protecting Kids: Developing a Vaccine for Respiratory Syncytial Virus

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Baby at the Doctor's OfficeVaccines are one of biomedicine’s most powerful and successful tools for protecting against infectious diseases. While we currently have safe and effective vaccines to prevent measles, mumps, and a great many other common childhood diseases, we still lack a vaccine to guard against respiratory syncytial virus (RSV)—a leading cause of pneumonia among infants and young children.

Each year, more than 2 million U.S. children under the age of 5 require medical care for pneumonia and other potentially life-threatening lower respiratory infections caused by RSV [1,2]. Worldwide, the situation is even worse, with more than 30 million infections estimated to occur annually, most among kids in developing countries, where as many as 200,000 deaths may result [3]. So, I’m pleased to report some significant progress in biomedical research’s long battle against RSV: encouraging early results from a clinical trial of an experimental vaccine specifically designed to outwit the virus.


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