[Policy Forum] Paying for future success in gene therapy

Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited neurodegenerative disease. For decades, gene therapy has tantalized us with such futuristic scenarios. However, these goals are now coming into focus, and it is the time to consider some of the consequences of success. Authors: Stuart H. Orkin, Philip Reilly

from Science: Current Issue http://ift.tt/1TLOXqV
via IFTTT

[Feature] Museum drawers go digital

No one knows exactly how many natural history specimens exist in museums and other research institutions worldwide, but some calculate it's on the order of 3 billion. In most cases, the displays seen by visitors make up a tiny slice of this treasure; museum curators estimate that more than 99% is stored away from the public gaze. Researchers have for decades used museum specimens to answer questions about how species diverge, where they move around the globe, and how they respond to changing conditions. But they have traditionally had to travel from museum to museum in person, or else request that the specimens be mailed to them. Now, even as museums struggle with funding woes that limit their activities, many around the world are working to put specimen photographs and related data online where anyone can view them. Until recently, these efforts were slow and painstaking, barely chipping away at the staggering amount of data in collections. Now, technological advances and innovative workflows are allowing institutions to think bigger, ushering in a new age of mass digitization. A new conveyor belt system at the Smithsonian Institution's National Museum of Natural History in Washington, D.C., will allow the digitization of 650,000 specimens within the period of a year, each one costing just $1. As digitization grows faster and cheaper, more governments and institutions are investing in it. Since 2011, the U.S. National Science Foundation has devoted $10 million per year to digitization efforts in nonfederal collections across the United States. But even with these new funding opportunities, museum officials and curators stress that there is still far too little money to make all specimens digital. Author: Nala Rogers

from Science: Current Issue http://ift.tt/1TIgkOa
via IFTTT

[Perspective] A brain conditioned for social defeat

Aggression is common in the animal kingdom, even though agonistic behaviors can lead to chronic stress or pain. So how does aggression remain conserved evolutionarily? In 1859, Darwin argued in his book On the Origin of Species that the conservation of any behavioral trait is ultimately explained by its necessity for survival and reproduction. To survive with limited resources, individuals express aggressive behaviors against competitors to pass on their genes. For social animals, dominance hierarchies establish rapidly (1), avoiding the cost of recurrent fighting within the group. Hierarchy formation and maintenance rely on the effect of prior experience (2). However, the underlying mechanisms and neural circuitry remain elusive. On page 87 of issue, Chou et al. (3) identify a key role for the dorsal habenula (dHb) region of the brain in zebrafish to determine who wins and who loses in a fight. This region is highly conserved across vertebrates, raising the possibility of manipulating neuronal circuits that govern innate social behaviors. Authors: Laura Desban, Claire Wyart

from Science: Current Issue http://ift.tt/1RNpI5p
via IFTTT

[In Depth] DNA helps build molecular libraries for drug testing

In the painstaking work of synthesizing vast numbers of compounds and identifying those that are the best candidate drugs, researchers have cultivated a capable new lab assistant: DNA. At a meeting last month just outside Boston, chemists and biologists discussed the promise of DNA-encoded chemical libraries (DELs), which rely on the unique talents of DNA to track, select, and even synthesize compounds that bind to enzymes, receptors, and other biological targets. The technology is allowing basic scientists and small companies to generate impressive libraries of molecules, on a scale once reserved for big pharma, and select from them the most useful compounds. Faster, cheaper, and more versatile than traditional screening methods, DELs are a potential game-changer for academics who want to probe the workings of biological molecules, and they have already yielded drug candidates entering clinical trials. Author: Trisha Gura

from Science: Current Issue http://ift.tt/1N5YzpS
via IFTTT

[In Depth] An obscure mosquito-borne disease goes global

A little-known virus called Zika has caused outbreaks in Pacific Ocean islands the past few years and has arrived in South America this year. Scientists predict it will spread far and wide in the Western Hemisphere, and perhaps in southern Europe as well, because the Aedes mosquitoes that transmit the virus are so widespread. Scientifically speaking, Zika virus is still largely terra incognita. Its symptoms, including rash, fatigue, headaches, muscle pains, and swollen and painful joints, appear to be generally mild, but during an outbreak in French Polynesia that started in 2013, some patients developed a serious neurological condition named Guillain-Barré syndrome. Although it is primarily spread by mosquitoes, some evidence suggests sexual transmission is possible as well. Author: Martin Enserink

from Science: Current Issue http://ift.tt/1LC2U1U
via IFTTT

[In Depth] Baby's leukemia recedes after novel cell therapy

A London baby with end-stage leukemia has received a remarkable new cancer treatment: off-the shelf T cells with several gene modifications. Doctors say it's too early to know whether she's cured, but the announcement advances a frontier in cancer immunotherapy, in which the body's immune system tackles the disease. For the past several years, researchers have been modifying T cells so they can attack leukemia, but the cells must be painstakingly isolated from the patients themselves and grown in a lab. Drug companies and many doctors dream of using off-the-shelf cells to make the therapy more like a regular drug. Now, by harnessing advances in genome editing to slice and dice genes in donor T cells, researchers have created a new type of cancer immunotherapy. Author: Jennifer Couzin-Frankel

from Science: Current Issue http://ift.tt/1MZAI08
via IFTTT

[Perspective] Restoring the sense of touch

Amputation of damaged tissue is one of the oldest surgical techniques, reaching prevalence in the 16th century (1). Improved emergency medicine has allowed more individuals to survive traumatic injuries as amputees, but prosthetic limbs remain the only means to restore any degree of function to these patients. Inadequate tactile feedback is a leading shortcoming of prosthetic limbs, but for artificial hands, just a few sensors that relay grasp pressure back to the user can provide the functionality needed to enable delicate tasks (2). In addition to improved motor control, sensory stimulation could alleviate phantom limb pain, which affects ~80% of amputees (2). On page 314 of this issue, Tee et al. (3) report a Digital Tactile System (“DiTact”) based on a low-power flexible organic transistor circuit that transduces pressure stimuli into oscillating signals like those generated by skin mechanoreceptors. Mammalian skin is a multilayered viscoelastic material that can stretch up to ~125% from its resting dimensions without any apparent loss in sensitivity to external stimuli such as pressure or temperature. Replicating skin mechanical and functional properties remains an elusive engineering challenge. Meanwhile, the rapidly expanding field of flexible electronics has made substantial strides, and complex circuits can now be produced on soft substrates. Advances in microcontact printing, inkjet deposition, and organic electronics have delivered stretchable and flexible, wearable, and even epidermal sensors (4–6). Authors: Polina Anikeeva, Ryan A. Koppes

from Science: Current Issue http://ift.tt/1VUQh89
via IFTTT

[Perspective] Yeast cell factories on the horizon

For thousands of years, yeast has been used for making beer, bread, and wine. In modern times, it has become a commercial workhorse for producing fuels, chemicals, and pharmaceuticals such as insulin, human serum albumin, and vaccines against hepatitis virus and human papillomavirus. Yeast has also been engineered to make chemicals at industrial scale (e.g., succinic acid, lactic acid, resveratrol) and advanced biofuels (e.g., isobutanol) (1). On page 1095 of this issue, Galanie et al. (2) demonstrate that yeast can now be engineered to produce opioids (2), a major class of compounds used for treating severe pain. Their study represents a tour de force in the metabolic engineering of yeast, as it involved the expression of genes for more than 20 enzymatic activities from plants, mammals, bacteria, and yeast itself. It clearly represents a breakthrough advance for making complex natural products in a controlled and sustainable way. Author: Jens Nielsen

from Science: Current Issue http://ift.tt/1KMBvin
via IFTTT