Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Columbia researchers identify drug as therapeutic candidate for spinal muscular atrophy

30.11.2004


Scientists develop data analysis tool, screen more than 47,000 compounds



Using a newly developed technology, a team of Columbia University researchers has uncovered that indoprofen, a nonsteroidal anti-inflammatory drug, may increase the production of a protein lacking in patients with spinal muscular atrophy (SMA), a fatal pediatric genetic disease.

It is the scientists’ hope that the discovery will lead to additional developments and even a treatment for SMA, a neurodegenerative disease that is the leading genetic killer of children younger than 2 in the United States and Western Europe. SMA has an incidence of 1 in 6,000 live births. Currently, no treatment exists. "Indoprofen is now both a chemical tool that researchers can use to study this disease and also a therapeutic candidate for these children," said Brent R. Stockwell, Columbia assistant professor in biological sciences and chemistry. This latest research was conducted at Stockwell’s lab, and is published in the November issue of the journal Chemistry & Biology in two separate articles: "Indoprofen Upregulates the Survival Motor Neuron Protein through a Cyclooxygenase-Independent Mechanism" and "A Flexible Data Analysis Tool for Chemical Genetic Screens."


Mitchell R. Lunn, Columbia senior research staff assistant and lead researcher on the study on indoprofen, which is structurally similar to ibuprofen, said that the next steps would be experiments to understand its mechanism of action, to deliver the drug to the spinal cord, and toxicity testing of related drug molecules. A clinical trial for its effectiveness in treating SMA could follow. It should be noted that the researchers found that ibuprofen and other nonsteroidal anti-inflammatory drugs had no effect on increasing the production of the protein lacking in individuals with SMA.

However, researchers uncovered the potential for indoprofen to increase the amount of Survival Motor Neuron (SMN) protein, the specific protein deficient in SMA patients. The results come from testing more than 47,000 compounds using a high-throughput chemical genetics approach. The data were studied with a robust analysis tool developed in the Stockwell Lab. This new software technology, known as Small Laboratory Information Management System (SLIMS), is freely available for academic use at http://slims.sourceforge.net.

"Our software package SLIMS enables researchers to analyze rapidly the mountains of data that result from chemical genetic screens," Stockwell said. For example, SLIMS is capable of organizing and "normalizing" raw data, meaning that it can "correct" for laboratory conditions such as humidity and temperature, which vary daily during the course of a high-throughput screening experiment. Additionally, SLIMS automatically identifies and corrects the systematic errors that are common with the robotic technologies used in screening.

Alissa Kaplan Michaels | EurekAlert!
Further information:
http://www.columbia.edu

More articles from Life Sciences:

nachricht A Map of the Cell’s Power Station
18.08.2017 | Albert-Ludwigs-Universität Freiburg im Breisgau

nachricht On the way to developing a new active ingredient against chronic infections
18.08.2017 | Deutsches Zentrum für Infektionsforschung

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Fizzy soda water could be key to clean manufacture of flat wonder material: Graphene

Whether you call it effervescent, fizzy, or sparkling, carbonated water is making a comeback as a beverage. Aside from quenching thirst, researchers at the University of Illinois at Urbana-Champaign have discovered a new use for these "bubbly" concoctions that will have major impact on the manufacturer of the world's thinnest, flattest, and one most useful materials -- graphene.

As graphene's popularity grows as an advanced "wonder" material, the speed and quality at which it can be manufactured will be paramount. With that in mind,...

Im Focus: Exotic quantum states made from light: Physicists create optical “wells” for a super-photon

Physicists at the University of Bonn have managed to create optical hollows and more complex patterns into which the light of a Bose-Einstein condensate flows. The creation of such highly low-loss structures for light is a prerequisite for complex light circuits, such as for quantum information processing for a new generation of computers. The researchers are now presenting their results in the journal Nature Photonics.

Light particles (photons) occur as tiny, indivisible portions. Many thousands of these light portions can be merged to form a single super-photon if they are...

Im Focus: Circular RNA linked to brain function

For the first time, scientists have shown that circular RNA is linked to brain function. When a RNA molecule called Cdr1as was deleted from the genome of mice, the animals had problems filtering out unnecessary information – like patients suffering from neuropsychiatric disorders.

While hundreds of circular RNAs (circRNAs) are abundant in mammalian brains, one big question has remained unanswered: What are they actually good for? In the...

Im Focus: RAVAN CubeSat measures Earth's outgoing energy

An experimental small satellite has successfully collected and delivered data on a key measurement for predicting changes in Earth's climate.

The Radiometer Assessment using Vertically Aligned Nanotubes (RAVAN) CubeSat was launched into low-Earth orbit on Nov. 11, 2016, in order to test new...

Im Focus: Scientists shine new light on the “other high temperature superconductor”

A study led by scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg presents evidence of the coexistence of superconductivity and “charge-density-waves” in compounds of the poorly-studied family of bismuthates. This observation opens up new perspectives for a deeper understanding of the phenomenon of high-temperature superconductivity, a topic which is at the core of condensed matter research since more than 30 years. The paper by Nicoletti et al has been published in the PNAS.

Since the beginning of the 20th century, superconductivity had been observed in some metals at temperatures only a few degrees above the absolute zero (minus...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Call for Papers – ICNFT 2018, 5th International Conference on New Forming Technology

16.08.2017 | Event News

Sustainability is the business model of tomorrow

04.08.2017 | Event News

Clash of Realities 2017: Registration now open. International Conference at TH Köln

26.07.2017 | Event News

 
Latest News

A Map of the Cell’s Power Station

18.08.2017 | Life Sciences

Engineering team images tiny quasicrystals as they form

18.08.2017 | Physics and Astronomy

Researchers printed graphene-like materials with inkjet

18.08.2017 | Materials Sciences

VideoLinks
B2B-VideoLinks
More VideoLinks >>>