DNAvaccine.com Presents: ‘Antibody Development for Human Therapy with DNA Immunisation’

Presentation Overview:
Dr. John Thompson will be discussing some of the key factors of antibody development for human therapy. For immunotherapeutic development, generating promising antibodies is a critical step as these must specifically recognise their target proteins in their native conformation. Aldevron proposes its proprietary GENOVAC Antibody Technology as an important tool to generate specific custom-made antibodies against native proteins. Several examples of the success of this technology will be shown including the recent collaboration with OMT to directly generate human antibodies. The presentation will be roughly 30 minutes in length and will include an open Q&A session for all attendees.

Title: Antibody Development for Human Therapy with DNA Immunisation
Date: Tuesday, April 29th, 2014
Time: 8:00 AM – 9:00 AM CDT
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Resource: ‘Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich’s ataxia’

Cardiac failure is the most common cause of mortality in Friedreich's ataxia (FRDA), a mitochondrial disease characterized by neurodegeneration, hypertrophic cardiomyopathy and diabetes1, 2, 3. FRDA is caused by reduced levels of frataxin (FXN), an essential mitochondrial protein involved in the biosynthesis of iron-sulfur (Fe-S) clusters4, 5, 6, 7, 8. Impaired mitochondrial oxidative phosphorylation, bioenergetics imbalance, deficit of Fe-S cluster enzymes and mitochondrial iron overload occur in the myocardium of individuals with FRDA9, 10, 11, 12. No treatment exists as yet for FRDA cardiomyopathy13, 14. A conditional mouse model with complete frataxin deletion in cardiac and skeletal muscle (Mck-Cre-FxnL3/L– mice) recapitulates most features of FRDA cardiomyopathy, albeit with a more rapid and severe course15, 16. Here we show that adeno-associated virus rh10 vector expressing human FXN injected intravenously in these mice fully prevented the onset of cardiac disease.
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Gene Therapy Successfully Regenerates an Old Organ Inside a Living Animal

In a landmark study sure to provoke interest, researchers from the University of Edinburgh have regenerated an aged organ — in vivo, inside a living animal — to its youthful state though noninvasive manipulation of genes. It’s a breakthrough that not only brings hope for a wide variety of age-related ailments, but which fundamentally challenges our idea of what aging is. This study treats the natural impacts of of time like symptoms of a disease — and by treating those symptoms it seems to have tracked the cells back to their pre-disease (youthful) state.
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Friedreich’s Ataxia: An Effective Gene Therapy in an Animal Model

The team led by Hélène Puccio, director of research for Inserm at the Institute of Genetics and Molecular and Cellular Biology in close collaboration with Patrick Aubourg's team has demonstrated, in the mice, the efficacy of gene therapy for treating the heart disease associated with Friedreich's ataxia, a rare hereditary neuro-degenerative disorder. The transfer, via a viral vector, of a normal copy of the gene deficient in patients, allowed to fully and very rapidly cure the heart disease in mice. These findings are published in Nature Medicine on 6 April, 2014.
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Clinical Trial Uses Gene Therapy to Target Mutations in Mitochondrial Genes

A multidisciplinary research team of scientists, clinicians and biostatisticians led by John Guy, M.D., professor of ophthalmology and director of the ocular gene therapy laboratory at the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, has pioneered a gene therapy approach for Leber Hereditary Optic Neuropathy (LHON), an inherited genetic disorder that causes rapid, permanent, and bilateral loss of vision in people of all ages, but primarily men ages 20-40. Guy’s preclinical research has been funded since 2007 by National Institutes of Health and National Eye Institute (NEI) grants totaling $6.1 million. A similar amount will fund a new five-year clinical trial that started April 1.
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Erasing a Genetic Mutation: Researchers Reverse a Liver Disorder in Mice by Correcting a Mutated Gene

Using a new gene-editing system based on bacterial proteins, MIT researchers have cured mice of a rare liver disorder caused by a single genetic mutation. The findings, described in the March 30 issue of Nature Biotechnology, offer the first evidence that this gene-editing technique, known as CRISPR, can reverse disease symptoms in living animals. CRISPR, which offers an easy way to snip out mutated DNA and replace it with the correct sequence, holds potential for treating many genetic disorders, according to the research team.
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FDA Grants Orphan Designation to Kite Cancer Immunotherapy

Kite Pharma Inc. (Kite), a clinical-stage biotechnology company focused on developing engineered autologous T cell therapy (eACT) products for cancer, announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted orphan drug designation for the company's lead investigational therapy, an autologous engineered T cell product that targets CD19 expression on B cell malignancies, for the treatment of diffuse large B cell lymphoma (DLBCL).
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Novel Cancer Vaccine Promising Against Ovarian Cancer and Mesothelioma

A protein engineered to combine a molecule targeting a tumor-cell-surface antigen with another protein that stimulates several immune functions was found to prolong survival in animal models of both ovarian cancer and mesothelioma. This novel approach to cancer immunotherapy may provide a new and cost-effective weapon against some of the most deadly tumors. Cancer immunotherapy uses strategies designed to induce the immune system to attack cancer cells.
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Scientists Build First Synthetic Yeast Chromosome

An international team of scientists has built a modified yeast chromosome from scratch, the latest step in the quest to make the world's first synthetic yeast genome, an advance that would lead to new strains of the organism to help produce industrial chemicals, medicines and biofuels. Instead of just copying nature, the team did extensive tinkering with their chromosome, deleting unwanted genes here and there. It then successfully incorporated the designer chromosome into living yeast cells, endowing them with new capabilities not found in naturally occurring yeast. "It is the most extensively altered chromosome ever built," said Jef Boeke of New York University's Langone Medical Center, who led the effort. The findings were published on Thursday in an online edition of the journal Science.
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Researchers Testing Gene Therapy to Thwart Effects of Multiple Sclerosis

In patients with multiple sclerosis, the body turns on itself, launching an immune system attack that destroys the coating around nerve fibers in the central nervous system, leaving them exposed like bare wires. Similar to exposed electrical lines, the unprotected fibers touch and short out, leading to the neurodegenerative effects that are a hallmark of multiple sclerosis. But what if doctors could stop the immune response that destroys the protective coating before the disease becomes debilitating? University of Florida researchers have received a $40,000 grant from the National Multiple Sclerosis Society to test a gene therapy technique in mice that aims to help the body not treat itself like a foreign invader — a process referred to as immune tolerance — in the earliest stages of multiple sclerosis. If the researchers can re-establish this tolerance, they could thwart the immune system attack, all with a technique that could be used on a wide number of patients.
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