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Man-made virus helps blind mice see the light

Posted on: 21 April 2015, source: horizon-magazine.eu
European scientists have reproduced the sensation of sight in blind mice by inserting light-reactive molecules into their optical nerve cells, and are now developing this treatment for use in humans. EU-funded researchers Dr Deniz Dalkara and Dr Jens Duebel at the Vision Institute in Paris, France, have designed a way of adding genes into a mouse's eye so that it responds to light independently of the natural mechinisms of the retina, the part of the eye concerned with light response. They have done this by using a light-sensitive molecule found in single-celled algae, which normally helps the algae swim towards light. The potential of this molecule to activate neurons in other species has been under investigation for years. The Vision Institute researchers have now found a way of capitalising on this, by using a virus to transport the algae-based molecule into the mouse's optical nerve cells.

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Researchers develop a harmless artificial virus for gene therapy

Posted on: 9 April 2015, source: Nanowerk.come
Researchers of the Nanobiology Unit from the UAB Institute of Biotechnology and Biomedicine, led by Antonio Villaverde, managed to create artificial viruses, protein complexes with the ability of self-assembling and forming nanoparticles which are capable of surrounding DNA fragments, penetrating the cells and reaching the nucleus in a very efficient manner, where they then release the therapeutic DNA fragments. The achievement represents an alternative with no biological risk to the use of viruses in gene therapy.

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FDA Approves Further Study Of Promising Gene Therapy HIV Treatment

Posted on: 19 March 2015, source: Towlerode
Experimental stem cell gene therapy that could act as functional cure for HIV infection has been approved by the Food and Drug Administration to move into early human test trials. Unlike other treatments that use healthy stem cells from uninfected donors, this form of therapy uses cells harvested from a positive person’s own body. The stem cells are genetically manipulated to develop into white blood cells that are missing the key cellular receptors that the HIV virus uses to insert its genetic code into healthy cells. The modification effectively models a HIV-positive person’s white blood cells after the cells of people who have a natural resistance to HIV.

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Will gene therapy make humans masters of own evolution?

Posted on: 12 March 2015, source: Discover Maganzine
Human genetic engineering is not new; it has been going on for a long, long time — naturally. Ancient viruses are really good at inserting themselves and modifying human gene code. Over millennia, constant infections would come to mean that 8 percent of the entire human genome is made up of inserted virus code. All this gene recoding of our bodies occurred under Darwin’s rules, natural selection and random mutation. But nonrandom, deliberate human genetic engineering is new, and it is a big deal.

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Paying for gene therapy: Are annuities the next big thing?

Posted on: 21 February 2015, source: St. Louis News
As U.S. drugmakers face growing resistance to the high price of cutting-edge treatments, a handful of companies is working on a new payment model that rewards them for the long-term performance of their medicines. The effort, industry executives say, is being led by firms developing so-called gene therapies, which aim to cure inherited diseases like hemophilia by “fixing” the single faulty gene responsible for the disorder. They include BioMarin Pharmaceutical Inc in San Rafael, Calif., and Sangamo BioSciences Inc in Richmond, Calif.
If these new hemophilia drugs and others like them succeed, a one-time infusion could replace the need for frequent, life-long injections of blood clotting proteins that can cost up to $300,000 a year for a single patient. These existing treatments, including Pfizer Inc.’s Xyntha and Baxter International’s Advate, are expected to command annual sales of more than $11 billion by 2016.

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Innovative nanoparticle gene therapy system eliminates cancerous brain cells

Posted on: 5 February 2015, source: Johns Hopkins
Despite decades of surgery, chemotherapy and radiation therapy treaments for glioma, a cure for this life-threatening brain cancer has remained elusive. In a study published on the website of the journal ACS Nano, BME Associate Professor Jordan Green and other Johns Hopkins researchers have successfully used compound-filled biodegradable nanoparticles to effectively kill brain cancer cells — and extend survival in rats.
The biodegradable nanoparticles filled with a DNA-encoded enzyme, herpes simplex virus type 1 thymidine kinase (HSVtk), proved to be potent in killing brain cancer cells. When researchers combined this with the compound ganciclovir, the loaded nanoparticles were 100 percent effective at killing glioma cells grown in laboratory dishes.

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U.K. Parliament approves controversial three-parent mitochondrial gene therapy

Posted on: 4 February 2015, source: Sciencemag
The United Kingdom’s House of Commons voted overwhelmingly today to allow British researchers to pursue a new fertility treatment that could prevent certain kinds of genetic diseases. The technique, called mitochondrial DNA replacement therapy, could allow women who carry disease-causing mutations in their mitochondrial genes to give birth to genetically related children free of mitochondrial disease.
The measure, which passed 382 to 128, has been controversial, especially because it would alter the DNA of an embryo in a way that could be passed on to future generations. Some scientists and nongovernmental organizations have argued that not enough is known about possible side effects of the technique to go forward in human patients. “We believe the House of Commons has made a serious mistake, which we hope does not have dire consequences,” said Marcy Darnovsky, executive director of the Center for Genetics and Society in Berkeley, California, in a statement.

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Controlled Gene Therapy May be Possible with New Method

Posted on: 11 January 2015, source: Bionews Texas
Gene therapy works by introducing genetic material (either DNA or RNA) into cells with a viral vector. This may be to replace a defective gene, but can also be to increase levels of a beneficial molecule that can compensate for the disease state. Most people have negative reactions when they think about viruses, but viral vectors are useful for injecting genetic material into cells. In research, and ultimately in the clinic, cells can then be controlled to churn out desired molecules for treating diseases. The use of viral vectors has held promise for gene therapy for many years, with the first gene therapy approved in the European Union in 2012. Despite gene therapy’s promise, researchers are still trying to overcome the limitations associated with this technology. Controlling where and how much of the gene is delivered to cells remains a formidable challenge. A new study, published in the journal Nucleic Acids Research in December 2014, may hold promise for enabling controlled gene therapy.

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Gene Therapy 3.0: Rise and fall and rise again of gene therapy–For real this time?

Posted on: 18 December 2014, source: Genetic Literacy Project
Gene therapy is back in the news, and in a big way as regular readers of the GLP would know. Studies involving the use of gene therapy are showing promising results for the cure of blood disorders, ‘bubble boy’ disease and HIV among others. Industry interest has also picked up and as positive results from clinical trials roll in, the market suddenly appears bullish on the future. But are we seeing enough to suggest that the technology is promising enough to make a major contribution to public health? Why does gene therapy have to be ‘back’ in the first place?
We offer a quick recap here, but for a more detailed read on the rise and fall (and rise again) of gene therapy, read this excellent narrative by Carl Zimmer in Wired or this comprehensive feature by Laura Cassiday in Nature. Things looked bright for gene therapy in the 90s when it promised to be a revolution that would let us move from just treating genetic diseases to curing them permanently. The big question surrounding gene therapy had always been how to effectively deliver the correct form of the gene into cells.

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Regulatory landscape of advanced-therapy medicinal products

Posted on: 14 December 2014, source: lexology.com
Advanced therapy medicinal products (ATMPs) are a category of innovative products comprising gene-therapy medicinal products (GTMPs),somatic cell-therapy medicinal products (sCTMP) and tissue-engineered products (TEPs). The main therapeutic areas are oncology and regenerative medicine, particularly in the field of cardiovascular conditions and haematology. Yet despite the harmonising EU legislation which has been in place since 2008, few products have reached the market in commercial form. In 2014 the European Commission issued a report on the implementation of ATMP legislation to date. On June 30 2014 the European Medicines Agency Committee for Advanced Therapies announced a public consultation on its revised guidance for ATMP classification. The consultation ended on October 31 2014.

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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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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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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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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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