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How to Make Biopharmaceuticals Quickly in Small Batches
Posted on by Dr. Francis Collins
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.
An Architectural Guide to the Nuclear Pore Complex
Posted on by Dr. Francis Collins
Credit: The Rockefeller University, New York
Sixty years ago, folk singer Pete Seeger recorded a song about helping those in need. The song starts like this: “Oh, had I a golden thread/And a needle so fine/I’d weave a magic strand/Of rainbow design.” In this brief animation, it seems like a golden thread and a needle are fast at work. But this rainbow design helps to answer a longstanding need for cell biologists: a comprehensive model of the thousands of pores embedded in the double-membrane barrier, or nuclear envelope, that divides the nucleus and its DNA from the rest of the cell.
These channels, called nuclear pore complexes (NPCs), are essential for life, tightly controlling which large macromolecules get in or out of the nucleus. Such activities include allowing vital proteins to enter the nucleus, blocking out harmful viruses, and shuttling messenger RNAs from the nucleus to the cytoplasm, where they are translated into proteins.
This computer simulation starts with an overhead view of the fully formed NPC structure. From this angle, the pore membrane (gray) appears to be at the base and is embroidered in four rings that are the channel’s main architectural support beams. There’s the cytoplasmic outer ring (yellow), the inner rings (purple, blue), the membrane ring (brown), and the nucleoplasmic outer ring (yellow). Each color represents different protein complexes, not rings per se, and the hole in the middle is the central channel through which molecules are transported. Filling the hole is a selective gating mechanism made of disordered protein (anchored to green) that helps to get the right molecules in and out.
Creative Minds: Using Machine Learning to Understand Genome Function
Posted on by Dr. Francis Collins
Anshul Kundaje / Credit: Nalini Kartha
Science has always fascinated Anshul Kundaje, whether it was biology, physics, or chemistry. When he left his home country of India to pursue graduate studies in electrical engineering at Columbia University, New York, his plan was to focus on telecommunications and computer networks. But a course in computational genomics during his first semester showed him he could follow his interest in computing without giving up his love for biology.
Now an assistant professor of genetics and computer science at Stanford University, Palo Alto, CA, Kundaje has received a 2016 NIH Director’s New Innovator Award to explore not just how the human genome sequence encodes function, but also why it functions in the way that it does. Kundaje even envisions a time when it might be possible to use sophisticated computational approaches to predict the genomic basis of many human diseases.
Happy New Year … and a Look Back at a Memorable 2015
Posted on by Dr. Francis Collins
A new year has arrived, and it’s going to be an amazing one for biomedical research. But before diving into our first “new science” post of 2016, let’s take a quick look back at 2015 and some of its remarkable accomplishments. A great place to reflect on “the year that was” is the journal Science’s annual Top 10 list of advances in all of scientific research worldwide. Four of 2015’s Top 10 featured developments directly benefited from NIH support—including Science’s “Breakthrough of the Year,” the CRISPR/Cas9 gene-editing technique. Here’s a little more on the NIH-assisted breakthroughs:
CRISPR Makes the Cut: I’ve highlighted CRISPR/Cas9 in several posts. This gene-editing system consists of a short segment of RNA that is attached to an enzyme. The RNA is preprogrammed to find a distinct short sequence of DNA and deliver the enzyme, which acts like a scalpel to slice the sequence out of the genome. It’s fast and pretty precise. Although CRISPR/Cas9 isn’t brand-new—it’s been under development as a gene-editing tool for a few years—Science considered 2015 to be “the year that it broke away from the pack.”
Creative Minds: Of Arsenic and Misfolded Proteins
Posted on by Dr. Francis Collins
John Hanna
Taking out the trash is a must in every household. Inside our cells, it’s also essential because if defective proteins are not properly disposed of, they can accumulate and make a mess of the cell’s inner workings, leading to health problems.
John Hanna, a physician-scientist at Brigham and Women’s Hospital, Boston, is on a quest to study the cell’s trash disposal system in greater detail. In particular, this 2014 NIH Director’s Early Independence awardee wants to learn more about how cells identify proteins that need to be discarded, how such proteins are steered towards the molecular garbage can, and how, when the process breaks down, neurodegenerative conditions, cancers, and other diseases can arise.
That’s a complex challenge, so Hanna will start by zeroing in on one particular component of cellular waste management—the component that clears out proteins damaged by arsenic. Although arsenic is notorious for being the poison of choice in countless true crime shows and mystery novels, this semi-metallic element is found naturally in soil, water, air, and some foods.
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