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Where in the world could the first CRISPR baby be born?

Posted on: 15 October 2015, source: Nature
They are meeting in China; they are meeting in the United Kingdom; and they met in the United States last week. Around the world, scientists are gathering to discuss the promise and perils of editing the genome of a human embryo. Should it be allowed — and if so, under what circumstances? A look at the legal landscape suggests where human genome editing might be used in research or reproduction.

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A Tale of Do-It-Yourself Gene Therapy

Posted on: 14 October 2015, source: Technology Review
An American woman claims she is the first to undergo gene therapy to reverse aging. Judge for yourself. Can aging be slowed by using gene therapy to make permanent changes to a person’s DNA? One Seattle-area woman says she has tried exactly that. Her claim has entangled some high-profile American academics in a strange tale of do-it-yourself medicine that involves plane flights to Latin America, an L.A. film crew, and what’s purported to be the first attempt to use gene therapy to forestall normal aging.

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Experimental gene therapy doubles glioblastoma survival rate

Posted on: 3 October 2015, source: UpdatedNews
An experimental gene therapy drug doubled the survival rate for glioblastoma, an aggressive brain cancer that kills two-thirds of patients within five years, researchers reported at a cancer conference. Typically, glioblastoma patients whose cancer comes back are expected to survive for weeks or months. Researchers reported the results of a Phase 2 clinical trial at the European Cancer Congress 2015 as Phase 3 of the trial begins.

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Promising Results on Gene Therapy for Congestive Heart Failure

Posted on: 10 September 2015, source: MD Magazine
San Diego-based Renova Therapeutics reported that its RT-100 gene therapy treatment for patients with reduced left-ventricular ejection fraction congestive heart failure (CHF) is proving safe and effective. The treatment involves delivering a therapeutic gene encoding adenylyl clyclase type 6 (AC6) directly into the heart via a modified adenovirus. It is a single-dose procedure done in an outpatient setting. Reporting on the results of a multi-center, placebo-controlled Phase 2 trial, company co-founder Kirk Hammond, MD, said the therapy showed a strong safety profile and clear dose response. The study assessed the safety of various doses of RT-100 in people with CHF.

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How CRISPR Could Change the World

Posted on: 15 August 2015, source: Tech Insider
Before 2012, researchers interested in studying how bacteria worked knew that microbes defended themselves against certain viruses using something they called a CRISPR/Cas system. There are a number of studies published in the early 2000s — starting in 2002 — discussing the presence of these systems and investigating how they work. There weren't many people studying that system then, especially compared to now, and those working in that area were doing what's called basic, or fundamental science, investigating something for the sake of curiosity or interest — not because they knew that research would have a practical or profitable result. They didn't know they were studying something that could be used to change the world, something that would give scientists the ability to rewrite DNA — the building blocks of life.

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Resurrection Of Extinct Virus Could Open The Doors To Gene Therapy Innovations

Posted on: 2 August 2015, source: Medical Daily
Gene therapy involves using viruses to replace missing or malfunctioning genes in order to treat some diseases, such as cancer. Experts believe this technique is at the forefront of medical treatment, but unfortunately it has one massive flaw: the immune system often destroys the viruses before they have the chance to deliver the therapy. According to a recent study, the way to get around this flaw could be by resurrecting an ancient virus.

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Positive Data Announced in Phase IIb Clinical Trial of Cystic Fibrosis Gene Therapy Manufactured by VGXI, Inc

Posted on: 11 July 2015, source: VGXI
Recently, the United Kingdom Cystic Fibrosis Gene Therapy Consortium (UKCFGTC) published the first clinical data from a Phase IIb, multi-dose clinical trial for cystic fibrosis. In this trial, patients received aerosolized DNA plasmid expressing Cystic Fibrosis Transmembrane Conductance Receptor (CFTR), manufactured at the VGXI cGMP production facility. This trial is the first to show that gene therapy can have a meaningful effect on the disease and benefit the lung function of patients with cystic fibrosis.

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Disruptive CRISPR gene therapy is 150 times cheaper than zinc fingers and CRISPR is faster and more precise

Posted on: 13 June 2015, source: Nextbigfuture
Biologists have long been able to edit genomes with molecular tools. About ten years ago, they became excited by enzymes called zinc finger nucleases that promised to do this accurately and efficiently. But zinc fingers, which cost US$5,000 or more to order, were not widely adopted because they are difficult to engineer and expensive, says James Haber, a molecular biologist at Brandeis University in Waltham, Massachusetts. CRISPR works differently: it relies on an enzyme called Cas9 that uses a guide RNA molecule to home in on its target DNA, then edits the DNA to disrupt genes or insert desired sequences. Researchers often need to order only the RNA fragment; the other components can be bought off the shelf. Total cost: as little as $30. “That effectively democratized the technology so that everyone is using it,” says Haber. “It's a huge revolution.”

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Chinese Gene-Editing Experiment Creeps Out Scientists

Posted on: 24 April 2015, source: Nature
Germ-line gene therapy: A group of scientists in China has just crossed one of biotechnology's red lines.
Chinese scientists have caused an uproar by trying to permanently edit the DNA of human embryos — created genetic changes that could be passed along from generation to generation. Their attempt didn't work very well, but the report, published in a small, online journal called Protein & Cell, has worried experts who have been watching out for such experiments. The motivation is to cure disease. In this experiment, the researchers were trying to correct defects in a gene called HBB that can cause a deadly blood disorder called beta-thalassemia. Gene therapy in adults and children is still experimental; the idea is to fix faulty disease-causing genes. But done in a very early embryo, the repair, called germline editing, would be permanent. It could also be passed along to future generations.

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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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