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Effectiveness of innovative gene therapy treatment demonstrated in canine model of DMD

Posted: November 13, 2014 at 6:42 pm

PUBLIC RELEASE DATE:

13-Nov-2014

Contact: Service de presse AFM-Tlthon gmonfort@afm-telethon.fr AFM-Tlthon @AfmPresse

Duchenne muscular dystrophy is the most common neuromuscular disease of children (affecting 1 boy in 3500-5000 births). It is caused by a genetic defect in the DMD gene residing on the X chromosome, which results in the absence of the dystrophin protein essential to the proper functioning of muscles.

The treatment being developed by researchers at Atlantic Gene Therapies, Gnthon and the Institute of Myology, is based on the use of an AAV vector (Adeno Associated Virus) carrying a transgene for the skipping of a specific exon which allows functional dystrophin production in the muscle of the patient.

Safety, efficacy and stability of the treatment in dogs

In GRMD (Golden Retriever Muscular Dystrophy) dogs the treatment aimed at skipping exons 6, 7 and 8 of the dystrophin gene. The product was given by loco-regional administration in the forelegs of 18 dogs who were followed for 3.5 months after injection. It was well tolerated by all treated dogs; no immune response against the synthesized dystrophin was observed. Exon skipping resulted in high levels of expression of dystrophin in the treated muscles. The results of this treatment also indicate that, once injected into the muscle tissue a prolonged and stable effect is produced over the observation time of the study and, unlike antisense oligonucleotides already used clinically for exon skipping, it does not need to be re- administered regularly. The synthesis of "new" dystrophin is dependent on the dose of vector injected: the higher the dose, the greater the exon skipping is effective. Muscle strength also increases with dose. 80% of muscle fibers expressed the "new" dystrophin at the highest dose. This is a very encouraging result because a minimum of 40% of dystrophin in muscle fibers is believed to be necessary for the muscle force to be significantly improved.

A phase I/II clinical trial phase

These results open the way for a phase I / II clinical trial by loco-regional administration in the upper limb of non-ambulatory Duchenne muscular dystrophy patients which are amenable to treatment by the specific skipping of exon 53. The regulatory toxicology and biodistribution studies have just ended and the filing of an application with regulatory authorities is planned for 2015.

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'Suck Me Shakespeer' Helps Revive Fortunes at Germany's Constantin Medien

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A politically-incorrect school-days comedy has helped revive fortunes at German mini-major Constantin Medien, which saw sales in its film division jump nearly 25 percent in the first nine months of this year to $279 million (223.6 million).

The results were helped in no small measure by home entertainment revenues from hit German-language comedy Suck Me Shakespeer. The division turned a $7.5 million (6 million) profit after losing $2.5 million (1.9 million) in the same period last year.

Constantin, already a major player on the German TV production scene, is looking to go global with its small-screen business and is planning TV series based on several of its film franchises, including YA fantasy series The Mortal Instruments and sci-fi horror Resident Evil.

Read more Germany's Constantin Media Results Slump as 'Mortal Instruments,' 'Enders Game' Disappoint

Overall, Constantin's nine month results showed a 14 percent jump in revenue to $451 million (361.6) and a net loss attributable to Constantin shareholders of $9.97 million (8 million). The company's sports division, which operates the network Sports1, saw some improvement over last year.

Constantin CEO Bernhard Burgener said the company had shown great progress, particularly in the digitalization of its traditional businesses.

Constantin boosted its full-year forecast and now expects losses attributable to shareholders of $8.7 million-$11 million (7 million - 9 million), down from $16 million-$18.7 million (13 million - 15 million). The company still expects to boost full-year revenue to $573 million-$623 million (460 million - 500 million).

Twitter: @sroxborough

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TRANSCENDING BORDERS , Stop Censorship – Video

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TRANSCENDING BORDERS , Stop Censorship

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HOW TO TRAIN YOUR DRAGON 2 | Unnecessary Censorship | Censored Parody Bleep Video – Video

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HOW TO TRAIN YOUR DRAGON 2 | Unnecessary Censorship | Censored Parody Bleep Video
This Week in Unnecessary Censorship, How to Train Your Dragon 2! Did you think this one was better than the first movie? Censorship tells the wrong story. Subscribe! comment! thanks for...

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Unnecessary Censorship – Dragon Age 2 (Censored Parody) – Video

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Unnecessary Censorship - Dragon Age 2 (Censored Parody)
Unnecessary Censorship - Dragon Age 2 (Censored Parody) Borderlands 2 is made by Bioware and owned by EA Games. Why not check out our other videos: Dragon Age Origins Part ...

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Turkey: Government Replaces Human Reproduction Images in School Textbooks With Pictures of Dolphins

Posted: at 6:42 pm

Pictures of animals such as dolphins are now been used to replace images of human genitalia in school textbooks(Ste Elmore/Flickr)

Pictures of human genitalia explaining the biological process of reproduction have been replaced with images of animals such as dolphins and ducklings in textbooks for primary school pupils in Turkey.

The replacement of the pictures sparked criticism, with many arguing the Islamist government is trying to promote conservative values, local Harriet Daily reported.

The move was also seen by some as a form of censorship applied by Turkish leaders.

Abdullah Tunal, a psychology expert who chaired the zmir branch of the teachers' union Eitim Sen, compared the 2014's textbooks with those used last year, and showed that the images in a chapter titled "Reproduction, Growth and Development in Living Beings" had been changed.

"In the past, the inner structure of genitalia was explained to children in appropriate ways for their development, just like heart and kidneys were pictured. But the sixth grade science and technology textbook has been seriously censored this year," Tunal said.

He added that the same section is now being taught "shortly, superficially and in a slapdash manner.

"We think the introductory information about a subject that is covered in more detail in the eighth class is not being taught healthily, as it has been censored.

"If scientific information is not taught at schools, a child's development can be harmed."

Censorship in Turkey

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Ron Paul: What The Elections Really Mean For Peace and Liberty – Video

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Ron Paul: What The Elections Really Mean For Peace and Liberty
What The Mid-Term Elections Really Mean For Peace and Liberty by Ron Paul Did the election last week really mean that much? I took to my Twitter account on T...

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GoldSeek Radio – Nov 7, 2014 [RON PAUL, LAURENCE KOTLIKOFF, & DAVID McALVENY] – Video

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GoldSeek Radio - Nov 7, 2014 [RON PAUL, LAURENCE KOTLIKOFF, DAVID McALVENY]
GoldSeek Radio #39;s Chris Waltzek talks to former Congressman, Dr Ron Paul http://www.ronpaulchannel.com/ Laurence J. Kotlikoff, a Professor of Economics at Boston Uni...

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Woolwich – Jews – AlJazeera – Ron Paul – Melting Pot – ISIS – Morris – Video

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Woolwich - Jews - AlJazeera - Ron Paul - Melting Pot - ISIS - Morris
Woolwich [hoax] Killer Called Ebola Jew No Ethnicity AlJazeera Doc on Israeli Shooting of USS Liberty Whitewashes USAF stand down Ron Paul On Dems Reps compete on foreign policy ...

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Rand Paul, advisers meet to strategize on a 2016 presidential bid

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Sen. Rand Paul, R-Kentucky, speaks at an election rally for Sen. Mitch McConnell, R-Kentucky, at Bowman Field November 3, 2014 in Louisville, Kentucky. Aaron P. Bernstein, Getty Images

In preparation for his all-but-announced 2016 presidential bid, Sen. Rand Paul, R-Kentucky, gathered staffers and political advisers from across the country for a meeting in Washington, D.C. on Wednesday.

The meeting, just eight days after midterm elections that saw Republicans seize the Senate majority and strengthen their grip on the House, was described as an opportunity for Paul and his advisers to pivot from the midterms to the next presidential cycle.

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"As I understand it, this meeting is to both literally and symbolically change focus after the November election," one person who was invited told National Journal, "and begin to take deliberate action toward a potential 2016 run."

"This is the come-to-Jesus before the planned launch," added another adviser.

Doug Wead, a presidential historian and adviser to Paul who attended Wednesday's meeting, told CBS News the gathering was "mind-blowing" in its organization.

"Have been a part of nine presidential campaigns in one way or another, this is the best organized, most intelligent I have seen yet," he said. "This crew has a sense of making history, of impacting the system, of ending corruption. They are young, attractive, smart, tech savvy, black, white, male, female. This event is supposed to be working on organizing the upcoming work, but this event itself is a model of efficiency."

The gathering of talent was perhaps the most obvious sign yet that Paul is on the cusp of announcing a presidential bid, but the Kentucky senator hasn't exactly made a secret of his ambition.

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