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Using Frogs to Tackle Kidney Problems

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Cilia

Credit: Vanja Krneta-Stankic and Rachel K. Miller, University of Texas Health Science Center at Houston

Many human cells are adorned with hair-like projections called cilia. Scientists now realize that these specialized structures play many important roles throughout the body, including directing or sensing various signals such as fluid flow. Their improper function has been linked to a wide range of health conditions, such as kidney disease, scoliosis, and obesity.

Studying cilia in people can be pretty challenging. It’s less tricky in a commonly used model organism: Xenopus laevis, or the African clawed frog. This image highlights a healthy patch of motile cilia (yellow) on embryonic skin cells (red) of Xenopus laevis. The cilia found in humans and all other vertebrates are built from essentially the same elongated structures known as microtubules. That’s why researchers can learn a lot about human cilia by studying frogs.


2018 Warren Alpert Foundation Prize Symposium

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I was deeply honored to be among the five recipients of the 2018 Warren Alpert Foundation Prize. All five recipients were recognized for their discoveries in contributing to the development of life-altering treatments for cystic fibrosis (CF). The other recipients were: Paul Negulescu, Vertex Pharmaceuticals, Boston; Bonnie Ramsey, University of Washington School of Medicine and Seattle Children’s Research Institute; Lap-Chee Tsui, The Academy of Sciences of Hong Kong; Michael Welsh, University of Iowa, Iowa City. We were recognized at an afternoon symposium titled Cystic Fibrosis: From Gene Discovery to Basic Biology to Precision Medicines. The symposium was held at Harvard Medical School, Boston, on October 4, 2018. The video posted here shows my presentation that afternoon. But if you would like to see more, there is a full video of this fantastic symposium.


How to Make Biopharmaceuticals Quickly in Small Batches

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Diagram showing three components of InSCyT system

Caption: InSCyT system. Image shows (1) production module, (2) purification module, and (3) formulation module.
Credit: Felice Frankel Daniloff, Massachusetts Institute of Technology, Cambridge

Today, vaccines and other protein-based biologic drugs are typically made in large, dedicated manufacturing facilities. But that doesn’t always fit the need, and it could one day change. A team of researchers has engineered a miniaturized biopharmaceutical “factory” that could fit on a dining room table and produce hundreds to thousands of doses of a needed treatment in about three days.

As published recently in the journal Nature Biotechnology, this on-demand manufacturing system is called Integrated Scalable Cyto-Technology (InSCyT). It is fully automated and can be readily reconfigured to produce virtually any approved or experimental vaccine, hormone, replacement enzyme, antibody, or other biopharmaceutical. With further improvements and testing, InSCyT promises to give researchers and health care providers easy access to specialty biologics needed to treat rare diseases, as well as treatments for combating infectious disease outbreaks in remote towns or villages around the globe.


Henrietta Lacks Memorial Lecture 2018

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Francis Collins standing with Roland Pattillo and Lacks family members

I enjoyed delivering the keynote address at the Henrietta Lacks Memorial Lecture 2018 on October 6 at Johns Hopkins University, Baltimore. Afterwards, I had the pleasure of meeting with members of the Lacks family and Roland Pattillo, Morehouse School of Medicine, Atlanta. From left to right is David Lacks, Jr., Devin Lacks, Alyana Rogers, Francis Collins, Roland Pattillo, Jabrea Rogers, Jeri Lacks Whye, and Dorian Lacks. Credit: Joshua Franzos


The Cancer Genome Atlas Symposium 2018

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