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Correcting the genetic error in sickle-cell disease might be as simple as amending text

Posted on: 15 November 2014, source: Nature
Tiny changes in DNA can have huge consequences. For years, scientists have been trying to 'fix' these mutations in the hope of treating and potentially curing some of humanity's most devastating genetic diseases. After some tragic early setbacks (see Nature 420, 116–118; 2002), techniques that allow precise genetic manipulation have created a surge of research.
Although most existing treatments for genetic diseases typically only target symptoms, genetic manipulation or 'gene therapy' goes after the cause itself. The approach involves either inserting a functional gene into DNA or editing a faulty one that is already there, so the conditions most likely to prove curable are those caused by a single mutation. Sickle-cell disease is a perfect candidate: it is caused by a change in just one amino acid at a specific site in the β-globin gene. This results in the production of abnormal haemoglobin proteins that cause the red blood cells that house them to twist and become sickle shaped. The distorted cells get sticky, adhere to each other and block blood vessels, preventing oxygenated blood from flowing through

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Popular lectures on gene therapy

Posted on: 6 November 2014, source: ieet.org
Maria Konovalenko and team put together a list of popular science video lectures on gene therapy – one of the most promising molecular medicine directions. What makes this approach different is that nucleic acid molecules, DNA and RNA, are used as therapeutic agents.

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Modified gene therapy shows promise in SCID-X1

Posted on: 9 October 2014, source: healio.com
A majority of boys with X-linked severe combined immunodeficiency experienced T-cell recovery and infection clearance after undergoing gene therapy with a self-inactivating gamma-retrovirus vector, according to study results. Salima Hacein-Bey-Abina, PharmD, PhD, of the department of biotherapy at Hôpital Necker – Enfants Malades in Paris, and colleagues sought to modify a Moloney murine leukemia virus-based gamma-retrovirus vector that expressed interleukin-2 receptor gamma-chain complementary DNA

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No-frills coats set a trend for designer viruses

Posted on: 28 August 2014, source: Chemistry World
Dutch scientists have built a simple model of viruses’ protective coats in an attempt to create viral mimics that could fight diseases, as opposed to causing them. Rather than copying natural proteins, Renko de Vries from Wageningen University and his team designed and built a three-part protein from scratch that self-assembles around DNA.
‘The protein is exceedingly simple in its primary and secondary structure, yet captures the essence of self-assembly for the tobacco mosaic virus,’ de Vries tells Chemistry World. This knowledge could enable superior vehicles for getting DNA and RNA into cells, for example for gene therapy, and templates for improved DNA machines. ‘You could probably do the same with supramolecular chemistry,’ de Vries adds, ‘but the protein approach has the beauty that you can expand in the direction of synthetic biology.’

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Retooling for Human Gene Therapy: New and Improved Adenoviral Vectors

Posted on: 21 August 2014, source: Sci-News.com
Dr Juliana Small of the University of Pennsylvania, Drs Raj Kurupati, Xianqyang Zhou and their colleagues from the Wistar Institute have developed a novel adenoviral vector for delivery of multiple transgenes.

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Gene correction technique could revolutionise treatment

Posted on: 6 August 2014, source: The Independent
Scientists have performed a “seamless” correction to a faulty gene behind an inherited form of anaemia using a revolutionary new technique in genome editing that could transform the treatment of many genetic diseases. Two mutations in the haemoglobin gene of a patient with beta thalassemia – which can cause severe anaemia – were corrected without any errors using the Crispr technique of genome editing, the researchers said. The experiment involved converting the patient’s skin cells into stem cells in the laboratory so that the faulty gene could be corrected before the stem cells were allowed to mature into red blood cells. Without the genome correction, the red cells would have become deformed and sickle-shaped as a result of the defective haemoglobin gene.

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New gene therapy may replace pacemaker implants

Posted on: 1 August 2014, source: WNCN
A new technology that allows genes to be injected into hearts with damaged electrical systems may replace the need for pacemaker implants in humans in the future. In the United States alone, there are more than 500,000 patients that get pacemaker implants annually. When the batteries on the Pacemakers run out in seven to 10 years, another surgery is required to implant a new device.

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Renewed hope for gene therapy in rare disease

Posted on: 1 July 2014, source: European Research Media Center
New virus serotypes are safely used as ‘DNA transporters’ to successfully deliver genes to deficient cells. Between 30 and 40 million people in Europe suffer from rare diseases—many of them children. As most of these diseases have genetic origins, gene therapy is a major hope for their future cure. Until now, however, there have been very few successful trials. Now, the EU-funded project AIPGENE, due to be completed in 2014, may have made significant progress in a gene therapy approach.
The project focussed on the genetic liver disorder, Acute Intermittent Porphyria (AIP). Through an early stage clinical trial, in phase I, it demonstrated the viability of a new approach, based on a so-called, adeno-associated vector (AAV). This is a ‘DNA transporter’ derived from a type of virus and carries the therapeutic gene to liver cells, known as hepatocytes.

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NIH Will No Longer Require Special Review for U.S. Gene Therapy Trials

Posted on: 26 May 2014, source: NIH
In a milestone for the field of gene therapy, the National Institutes of Health (NIH) will no longer subject all proposed gene therapy clinical trials to review by a special federal advisory committee. “Given the progress in the field, I am confident that the existing regulatory authorities can effectively review most gene transfer protocols and that a streamlined process will reduce duplication and delays in getting gene transfer trials initiated,” said NIH Director Francis Collins in a statement today. Instead, the 40-year-old Recombinant DNA Advisory Committee (RAC) will review only a few trials that pose special risks.

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Gene therapy injections: Future obesity cure?

Posted on: 14 May 2014, source: MetroNews
An injection that promises to end obesity seems like the type of claim found only on obnoxious flashing web ads, but it’s entirely plausible that one day we will be able to treat this common problem with just the prick of a needle, according to Jason Dyck, a researcher at the University of Alberta. Two years ago, Dyck and his colleagues published a paper in the journal Nutrition and Diabetes that concluded an injectable adiponectin gene therapy reduced fat and improved insulin sensitivity in mice, despite the fact the test animals were being fed a high-fat diet.

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FDA Grants Breakthrough Status to Gene Therapy for First Time

Posted on: 11 April 2014, source: Bio-ITWorld
Celladon today became the first company to receive breakthrough status for a gene therapy treatment in development. The treatment, MYDICAR, is intended to reduce the risk of heart failure in patients with a deficiency of the enzyme SERCA2a. The FDA made its decision to grant breakthrough status to MYDICAR on the basis of Celladon's Phase 1 trial of 39 systolic dysfunction patients. The study reported that subjects receiving high doses of MYDICAR experienced fewer heart failure events than subjects given a placebo, by a factor of more than 80%, and that this reduction was sustained for three years after treatment. No safety issues were reported in this initial trial. Breakthrough status, which is reserved for treatments targeting life-threatening diseases that show significant improvements over the standard of care, allows closer communication with the FDA and potentially an accelerated approval process.

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Stem cell gene therapy: Chances and risks

Posted on: 20 March 2014, source: HealthCanal
Physicians and scientists from Munich and Heidelberg have now proven the long-term effectiveness of stem cell gene therapy, based on a study of patients from the first clinical trial worldwide using gene therapy to treat Wiskott-Aldrich syndrome. Yet several years after the therapy, the researchers also observed an increased incidence of acute leukemia among the patients.

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International Gene Therapy Consortium (IGTC) is an important step in 2014

Posted on: 13 March 2014, source: International Gene Therapy Consortium
Established at the meeting of the American Society of Gene and Cell Therapy (ASCGT) in May 2013, the Consortium, which is comprised of leading scientists and researchers from more than 50 locations worldwide, has created a website where patients can interact with scientists from the organization. An online course of gene therapy was created with the collaboration of many researchers to facilitate the teaching of this discipline to students in science and medicine. Patients can also ask questions about various genetic diseases. There will also be a virtual meeting place and a forum for discussion on their diseases.

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University of Westminster develops groundbreaking method to test hepatitis C cure

Posted on: 17 February 2014, source: University of Westminster
Researchers at the University of Westminster have developed a groundbreaking method which can be used to test a new innovative cure for hepatitis C, a liver disease caused by the hepatitis C virus (HCV). The cure is the first of its kind ever to be tested in humans and comes in the form of a drug based on gene therapy which is under development by the Australian company Benitec Biopharma. Around 150 million people worldwide are infected with hepatitis C, and more than 350,000 people die every year from hepatitis C related liver diseases. Hepatitis C is one of the leading causes of liver cirrhosis and cancer, and one of the most common and seriously infectious conditions in the world.

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Nanotech robots deliver gene therapy through blood

Posted on: 29 January 2014, source: WorldBulletin
U.S. researchers have developed tiny nanoparticle robots that can travel through a patient's blood and into tumors where they deliver a therapy that turns off an important cancer gene. The finding, reported in the journal Nature on Sunday, offers early proof that a new treatment approach called RNA interference or RNAi might work in people.

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Gene therapy promising in Parkinson's patients

Posted on: 14 January 2014, source: BioNews
Patients in a clinical trial to treat Parkinson's disease with a form of gene therapy have showed signs of significant improvements in their motor-function, according to a report published in the Lancet. Fifteen advanced-stage Parkinson's patients (three from the UK and 12 from France) were followed up a year after being injected with low, mid and high doses of a modified virus containing genes required for brain cells to produce dopamine, as part of the phase I/II study. A lack of dopamine causes patients with Parkinson's to experience tremors and difficulty in coordinating their movement. The researchers observed that after receiving the treatment the patients' scores on movement tests improved on average by 30 percent. The patients also reported having a better quality of life. Although the patients showed some side effects, overall the treatment was found to be safe and no serious side effects were observed.

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Gene therapy scores in fight against leukemia

Posted on: 23 December 2013, source: JapanNews
In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients’ blood cells into soldiers that seek and destroy cancer. A few patients with one type of leukemia were given this one-time, experimental therapy several years ago and some remain cancer-free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.

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Explainer: what is gene therapy?

Posted on: 28 November 2013, source: Science Alert
Every now and again you might read about gene therapy and efforts to correct serious genetic diseases. But I’m betting that very few readers have had gene therapy, nor have they ever met anyone who has, nor will they ever meet that many. The reasons are simple – while these procedures are possible in theory, in practice, it has proved remarkably difficult to insert new genetic materials into human patients and ensure that the new genes are sustainably expressed. The difficulties encountered highlight the fact that we do not yet have enough fundamental knowledge about gene transfer and control. And what’s more, gene therapy is hugely expensive.

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Q&A: Gene Therapy the Fix for Faulty DNA

Posted on: 27 November 2013, source: ModVive.com
Inside (almost) all of our cells are 23 pairs of chromosomes, long strands of DNA that are packaged tightly together. Each chromosome houses hundreds of genes, each of which is the blueprint for at least one protein. Little messengers called mRNA make copies of the gene blueprints, edits them and carry them to “builders” called ribosomes. The ribosomes read the copy and translate it into long chains of amino acids that are then folded into unique shapes. These globs of amino acids are proteins, that travel throughout our entire body and are in some way vital for everything that happens inside of us. For example, helicase and chromatin are proteins essential to cell division, what allows us to grow and heal, and immunoglobulin, also called antibodies, protect us from infections.

What happens when something goes wrong with our DNA and thus our proteins?

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UK's first gene therapy trial for patients with chronic heart failure begins at GJNH

Posted on: 21 November 2013, source: News-Medical
The UK’s first gene therapy trial for advanced heart failure, CUPID 2, has officially begun at the Golden Jubilee National Hospital (GJNH); with the first candidate recently being administered with a dose of the MYDICAR treatment. The GJNH, home of the Scottish National Advanced Heart Failure Service (SNAHFS), announced in April 2013 that it would be participating in the international trial, helping gather a total of 200 patients from 50 institutions worldwide, to determine the effectiveness of the gene therapy treatment, MYDICAR, in advanced heart failure patients.

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Gene therapy company launched

Posted on: 1 November 2013, source: Boston Globe
Fidelity Biosciences, a venture capital firm that is a subsidiary of Fidelity Investments, and REGENX Biosciences, announced the formation of Dimension Therapeutics, a Cambridge gene therapy company focused on developing treatments for rare diseases such as hemophilia. Dimension has completed an undisclosed Series A financing that was led by Fidelity Biosciences.

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Several gene therapies are or will soon be in late-stage human trials

Posted on: 2 October 2013, source: NextBigFuture
Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials. Experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

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Vaccine appears to clear HIV-like virus in monkeys

Posted on: 14 September 2013, source: Science Recorder
Researchers at Oregon Health and Science University (OHSU) have developed a candidate vaccine that appears to completely clear an AIDS-causing virus from the body. Developed at OHSU’s Vaccine and Gene Therapy Unit, the vaccine is now being tested through a non-human primate form of HIV, called simian immunodeficiency virus (SIV). It is hoped that an HIV-form of the vaccine will soon be tested on humans.

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Gene therapy repairs heart attack damage

Posted on: 29 August 2013, source: BioNews
Researchers have concocted a 'gene cocktail' that may help to turn scar tissue into functioning cardiac cells after a heart attack. Scar tissue often forms following a heart attack in the area that was most affected. The result is a decreased ability of the heart to beat properly and can eventually lead to heart failure. The scientists, from the Gladstone Institutes in San Francisco, USA, are working with cells called fibroblasts, which make up about half of all heart cells. They are aiming to reprogram the function of these fibroblasts from primarily providing structural support to beating heart cells by injecting a cocktail of genes.

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First Gene Therapy For Rett Syndrome, An Autism Spectrum Disorder

Posted on: 21 August 2013, source: Medical Daily
Scientists in Oregon have developed a new gene therapy for Rett Syndrome, the most disabling of the autism spectrum disorders. Field-tested in mice, it is the first therapy to reverse the course of the disorder in a fully symptomatic model, which represents an important step towards clinical application. Nearly one in 10,000 kids are born each year with Rett syndrome. Neurological and behavioral features, like loss of speech, immobility, and seizures, typically appear after six months of age. The most frequent cause is the mutation of an X chromosome gene called MECP2, which explains why girls are primarily affected by the condition.

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Rules for Gene-therapy Vectors

Posted on: 14 August 2013, source: Health News
Rice University researchers are making strides toward a set of rules to custom-design Lego-like viral capsid proteins for gene therapy. A new paper by Rice scientists Junghae Suh and Jonathan Silberg and their students details their use of computational and bioengineering methods to combine pieces of very different adeno-associated viruses (AAVs) to create new, benign viruses that can deliver DNA payloads to specific cells. The research appears this month in the American Chemical Society journal ACS Synthetic Biology.

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Wiskott-Aldrich Syndrome: Lentivirus Gene Therapy Helps Three Kids

Posted on: 14 July 2013, source: Science 2.0
An international team that developed a new gene therapy approach to treatment of Wiskott-Aldrich Syndrome, a fatal inherited form of immunodeficiency has reported results. Wiskott-Aldrich Syndrome is a disorder that weakens the body's immune system. It is caused by a mutation in a gene that encodes the protein WASP. The most often used therapy is a bone marrow or stem cell transplant from a matching donor, often a sibling or relative. It can be curative for some patients, mostly those who have a strongly matching donor.

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WADA Symposium Covers Broad Spectrum of Gene Doping, and Potential for Misuse of Cell Therapy in World of Sport

Posted on: 7 June 2013, source: WADA
Leading experts in gene therapy, scientists from the field of anti-doping, and sport ethicists joined WADA’s Gene Doping Panel at the Agency’s Fourth Gene and Cell Doping Symposium, held June 5-6 in Beijing to review recent achievements in the field and share the most promising approaches in doping detection under development. Organized with the support of the China Anti-Doping Agency (CHINADA) and Beijing Olympic City Development Association (BODA), the Symposium attracted more than 70 international participants. Sixteen invited speakers included international experts who either study the latest advances in the field of gene therapy and gene doping detection methods, or are involved in related WADA-funded research projects. The 2013 Symposium was the first to dedicate a formal session to cell therapy and doping.

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Public consultation on advanced therapy medicinal products: A summary of the responses

Posted on: 30 May 2013, source: European Commission
The European Commission should prepare a report on the application of Regulation (EC) No 1394/2007 on advanced therapy medicinal products. With a view to prepare this report, the Commission services launched a public consultation on 20 December 2012. Stakeholders were invited to provide their views on the requirements to obtain a marketing authorisation for an ATMP (including for combined ATMPs), on the application of the so-called hospital exemption, on the incentives provided for in the ATMP Regulation, as well as on the scope thereof and its adaptation to technical progress. The number of contributions received was 63.

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Scientists reveal inside story of European regulatory review and approval of Glybera

Posted on: 23 May 2013, source: News Medical
The scientists who led the team that developed Glybera, the first gene therapy drug approved for use in the Western world, provide a fascinating first-person account of their pioneering work in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. In addition, an in-depth Review revealing the inside story of the European regulatory review and approval of Glybera—chock full of twists and turns, politics, and intrigue— and critiquing the groundbreaking drug's path to the marketplace appears in Human Gene Therapy Clinical Development. Both articles are available free on the Human Gene Therapy website.

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