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Category Archives: Transhuman News

Gene therapy may be possible cure for Hurler syndrome: Mouse Study

Posted: February 5, 2014 at 11:42 am

Researchers used blood platelets and bone marrow cells to deliver potentially curative gene therapy to mouse models of the human genetic disorder Hurler syndrome -- an often fatal condition that causes organ damage and other medical complications.

Scientists from Cincinnati Children's Hospital Medical Center and the National Institute of Neurological Disorders and Stroke (NINDS) report their unique strategy for treating the disease the week of Feb. 3-7 in Proceedings of the National Academy of Sciences (PNAS).

Researchers were able to genetically insert into the cells a gene that produces a critical lysosomal enzyme (called IDUA) and then inject the engineered cells into mice to treat the disorder. Follow up tests showed the treatment resulted in a complete metabolic correction of the disease, according to the authors.

"Our findings demonstrate a unique and somewhat surprising delivery pathway for lysosomal enzymes," said Dao Pan, PhD, corresponding author and researcher in the Division of Experimental Hematology and Cancer Biology at Cincinnati Children's. "We show proof of concept that platelets and megakaryocytes are capable of generating and storing fully functional lysosomal enzymes, which can lead to their targeted and efficient delivery to vital tissues where they are needed."

The mice tested in the study modeled human Hurler syndrome, a subset of disease known as mucopolysaccharidosis type I (MPS I), one of the most common types of lysosomal storage diseases. MPS I is a lysosomal storage disease in which people do not make an enzyme called lysosomal alpha-L-iduronidase (IDUA).

IDUA helps break down sugar molecules found throughout the body, often in mucus and fluids around joints, according to the National Library of Medicine/National Institutes of Health. Without IDUA, sugar molecules build up and cause organ damage. Depending on severity, the syndrome can also cause deafness, abnormal bone growth, heart valve problems, joint disease, intellectual disabilities and death.

Enzyme replacement therapy can be used to treat the disease, but it is only temporary and not curative. Bone marrow transplant using hematopoietic stem cells also has been tested on some patients with mixed results. The transplant procedure can carry severe risks and does not always work.

Pan and her colleagues -- including Roscoe O. Brady, MD, a researcher at NINDS -- report that using platelets and megakaryocytes for gene therapy is effective and could reduce the risk of activating cancer-causing oncogenes in hematopoietic stem cells.

The authors said tests showed that human megakaryocytic cells were capable of overexpressing IDUA, revealing their capacity for potential therapeutic benefit. While engineering megakaryocytes and platelets for infusion into their mouse models of Hurler, the scientists report they were able to release IDUA directly into amply sized extracellular spaces or inside micro-particles as the cells matured or activated. The cells were able to produce and package large amounts of functional IDUA and retained the capacity to cross-correct patient cells.

After infusing mouse models of Hurler with the genetically modified cells, researchers said this led to long-term normalization of IDUA levels in the animal's blood with versatile delivery routes and on-target preferential distribution to the liver and spleen. The treatment led to a complete metabolic correction of MPS I in most peripheral organs of the mice.

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New Fruitfly Sleep Gene Promotes the Need to Sleep

Posted: at 11:42 am

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Newswise PHILADELPHIA All creatures great and small, including fruitflies, need sleep. Researchers have surmised that sleep in any species -- is necessary for repairing proteins, consolidating memories, and removing wastes from cells. But, really, sleep is still a great mystery.

The timing of when we sleep versus are awake is controlled by cells in tune with circadian rhythms of light and dark. Most of the molecular components of that internal clock have been worked out. On the other hand, how much we sleep is regulated by another process called sleep homeostasis, however little is known about its molecular basis.

In a study published in eLIFE, Amita Sehgal, PhD, professor of Neuroscience at the Perelman School of Medicine, University of Pennsylvania, and colleagues, report a new protein involved in the homeostatic regulation of sleep in the fruitfly, Drosophila. Sehgal is also an investigator with the Howard Hughes Medical Institute (HHMI).

The researchers conducted a screen of mutant flies to identify short-sleeping individuals and found one, which they dubbed redeye. These mutants show a severe reduction in the amount of time they slumber, sleeping only half as long as normal flies. While the redeye mutants were able to fall asleep, they would wake again in only a few minutes.

The team found that the redeye gene encodes a subunit of the nicotinic acetylcholine receptor. This type of acetylcholine receptor consists of multiple protein subunits, which form an ion channel in the cell membrane, and, as the name implies, also binds to nicotine. Although acetylcholine signaling -- and cigarette smoking -- typically promote wakefulness, the particular subunit studied in the eLIFE paper is required for sleep in Drosophila.

Levels of the redeye protein in the fly oscillate with the cycles of light and dark and peak at times of daily sleep. Normally, the redeye protein is expressed at times of increasing sleep need in the fly, right around the afternoon siesta and at the time of night-time sleep. From this, the team concluded that the redeye protein promotes sleep and is a marker for sleepiness suggesting that redeye signals an acute need for sleep, and then helps to maintain sleep once it is underway.

In addition, cycling of the redeye protein is independent of the circadian clock in normal day:night cycles, but depends on the sleep homeostat. The team concluded this because redeye protein levels are upregulated in short-sleeping mutants as well as in wild-type animals following sleep deprivation. And, mutant flies had normal circadian rhythms, suggesting that their sleep problems were the result of disrupted sleep/wake homeostasis.

Ultimately the team wants to use the redeye gene to locate sleep homeostat neurons in the brain. We propose that the homeostatic drive to sleep increases levels of the redeye protein, which responds to this drive by promoting sleep, says Sehgal. Identification of molecules that reflect sleep drive could lead to the development of biomarkers for sleep, and may get us closer to revealing the mystery of the sleep homeostat.

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New Fruitfly Sleep Gene Promotes the Need to Sleep

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ND Farm Bureau says it's 'lost confidence' in ag commissioner, backs challenger Estenson

Posted: at 11:42 am

Judy Estenson and her husband, Hal

With her husband Hal at her side, Judy Estenson announces her intentions to run for North Dakota commissioner of agriculture during a press conference at the Ramada Plaza Suites, Tuesday, January 4, 2014. Darren Gibbins / The Forum

Doug Goehring

BISMARCK The North Dakota Farm Bureau is supporting a challenger for the Republican endorsement for state agriculture commissioner, saying Tuesday it has lost confidence in incumbent Commissioner Doug Goehring one day after Goehring emailed supporters defending his record and explaining a politically incorrect statement he made to his staff.

Judy Estenson announced her candidacy at news conferences in Bismarck and Fargo, and a Farm Bureau official was on hand to distribute a statement explaining why it is supporting her over Goehring, a Farm Bureau member and former vice president of the organization.

Estenson will run as a Republican, setting up an intraparty challenge in the 2014 election as well as Goehrings fight with an organization that backed his campaign in 2010.

Goehring said in an interview Tuesday that while people have a right to challenge him for the endorsement, its unfortunate that its being done with innuendo and misrepresentation of the facts, untruths and character assassination.

It does hurt that theres a faction in Farm Bureau that I thought were my friends that did this, he said.

Public records show North Dakota Farm Bureau President Doyle Johannes requested state files involving sexual harassment claims and hostile workplace environment claims against Goehring.

Public records of the investigation show Goehring reportedly introduced one new female employee as a babe in the woods and referred to a group of women some of them employees as a harem. Both incidents happened in the summer of 2012.

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ND Farm Bureau says it's 'lost confidence' in ag commissioner, backs challenger Estenson

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North Dakota Farm Bureau says it's 'lost confidence' in ag commissioner, backs challenger Estenson

Posted: at 11:42 am

Doug Goehring

North Dakota Ag Commissioner

Estenson will run as a Republican, setting up an intraparty challenge in the 2014 election as well as Goehrings fight with an organization that backed his campaign in 2010.

It does hurt that theres a faction in Farm Bureau that I thought were my friends that did this, he said.

Public records show North Dakota Farm Bureau President Doyle Johannes requested state files involving sexual harassment claims and hostile workplace environment claims against Goehring.

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North Dakota Farm Bureau says it's 'lost confidence' in ag commissioner, backs challenger Estenson

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Live Review: Bachelors of Art, Megan Jean and the KFB at Art Bar

Posted: at 11:42 am

The Playlist

Though They Peddled Different Styles, Both Bands Succeeded Through Vocal Excellence

Bachelors of Art, courtesy photo

This is going to sound sexist and politically incorrect, but it has to be said, and it really doesnt come out right any other way nothing rocks more than chicks in rock bands. Friday nights lineup at Art Bar proved this in triplicate with three bands fronted and dominated by women.

Megan Jean and the KFB is a duo of Jean and her husband Byrne Klay, but they filled up the room and opened the show with the energy of a much larger ensemble. Jean takes on the lions share of performing duties, employing washboard, snare, tambourine, and an inventively placed bass drum; its on the floor behind her, and she uses her heel on the pedal. She pounded out the low end throughout the set. Klays role is that of color commentator, plinking a banjo tied into some electric effects pedals for an unlikely melodic accompaniment.

Its unusual to see the initial act on a mult-band lineup fill the room, but Jean is that rarity of a singer whos as savvy an entertainer as she is a vocalist, coaxing the crowd toward the stage with comedic asides and her expressive face. Then theres that voice, a foghorn of emotional truth. Jeans a rootsier version of P.J. Harveys militant blueswoman stance, powerful and occasionally profound with a sometimes chilling tone that gets under your skin and crawls around as it rips a room full of listeners apart.

Bachelors of Arts fans are an admittedly aging force of former local scenesters who, if I were being a cynic about the reunited bands occasional live shows, are merely rehashing their glory days to the soundtrack of those late night gigs at the Beat or Rockafellas. Saving them and the rest of the crowd from what could have been a simple trip down Nostalgia Lane was the tremendous performance put on by the band, which in its initial incarnation morphed from an artsy gothic rock act into a hard-hitting goth-metal powerhouse, stripping its name down to the essential initials as B.O.A.

Vocalist Robin Wilson was always the focal point of B.O.A., and rightly so. Her instrument is a throaty roar that she exercises complete control over even 20 years down the road. Id venture to say that she looks and sounds even better today than she did at the bands peak. Their sound may be a little dated at this late juncture, with guitarist Tom Alewines delay pedal evoking The Edges early work with U2 while the throbbing pulse provided by bassist Jay Matheson and drummer Blake Liles is so much like Love-era Cult that the closing rendition of that bands Rain is just about the perfect cover choice. This didnt make B.O.A.s first real club set in two decades any less impressive, however, and both Megan Jean and her counterpart from headliners Handsome Devil watched slackjawed at Wilsons seemingly effortless performance.

Following two talented and charismatic singers would be a tall order for anyone, and Handsome Devils Molly Williamson admitted as much from the start of the groups set with the query, Yall dont want to hear another band that can hit every note, do you? The band then launched into a set of endearingly sloppy pop-punk anthems. Guitarist Jesse Ferrari handles more of the lead vocals than Williamson, but it is her smiling, sunny demeanor on stage that makes Handsome Devil more fun than ferocious. This is punk rock for when youre in a good mood. Shades of The Clash, 7 Seconds, and other predictable touchstones littered the songs, but it was hard not to get carried along in the enthusiasm despite the missed notes, making this the exclamation point to an evening of enthusiastic performances.

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Live Review: Bachelors of Art, Megan Jean and the KFB at Art Bar

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nternet zgrlkleri Sansr Aacnda Asld / Internet Freedom Hanged on Censorship Tree – Video

Posted: at 11:42 am

nternet zgrlkleri Sansr Aacnda Asld / Internet Freedom Hanged on Censorship Tree
http://www.korsanparti.org Korsan Parti Trkiye, sansr, gzetleme, basklara kar eylem arsnda... #internetimedokunma #sansuredurde Pirate Party of Turkey cal...

By: Korsan Parti

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nternet zgrlkleri Sansr Aacnda Asld / Internet Freedom Hanged on Censorship Tree - Video