New Age of Genome Editing Could Lead to Cure for Sickle Cell Anemia

UNSW Australia researchers have shown that changing just a single letter of the DNA of human red blood cells in the laboratory increases their production of oxygen-carrying haemoglobin -- a world-first advance that could lead to a cure for sickle cell anemia and other blood disorders. The new genome editing technique, in which a beneficial, naturally-occurring genetic mutation is introduced into cells, works by switching on a sleeping gene that is active in the womb but turned off in most people after birth. "An exciting new age of genome editing is beginning, now that single genes within our vast genome can be precisely cut and repaired," says study leader, Dean of Science at UNSW, Professor Merlin Cr
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Gene Therapy Comes of Age

It seldom happens that a premature shoot of genius ever arrives at maturity. One such shoot, gene therapy, appears to be one of the exceptions. Gene therapy, which showed early promise as a means of replacing defective or missing genes, is branching out. And it may yet produce abundant and diverse fruits. For example, gene therapy is being cultivated in cardiovascular applications, which are relevant to large, broad-based patient populations. Gene therapy approaches to cardiovascular and other diseases are being tended in billion-dollar collaborations, and they are being evaluated in late-stage clinical trials. Current gene therapy products in development utilize a variety of viral vectors. Some of them integrate into the host cell genome to achieve long-term protein expression; others do not, aiming for only transient expression of a therapeutic product.
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Experimental Gene Therapy Holds Promise Against Metastatic Prostate Cancer

Even with the best available treatments, the median survival of patients with metastatic, hormone-refractory prostate cancer is only two to three years. Driven by the need for more effective therapies for these patients, researchers at VCU Massey Cancer Center and the VCU Institute of Molecular Medicine (VIMM) have developed a unique approach that uses microscopic gas bubbles to deliver directly to the cancer a viral gene therapy in combination with an experimental drug that targets a specific gene driving the cancer's growth. Recently published in the journal Oncotarget, this new study is the most recent in a long line of studies led by Paul B. Fisher, M.Ph., Ph.D., investigating the use of viral gene therapy to treat a variety of cancers. The treatment strategy uses a novel "cancer terminator virus" (CTV), which replicates exclusively in cancer cells delivering the cancer-specific, toxic cytokine gene mda-7/IL-24 directly to the tumor. The researchers added an experimental drug known as BI-97D6, which targets MCL-1 and other members of the Bcl-2 gene family that protect cancer cells from therapeutic agents, resulting in enhanced prostate cancer cell death while sparing healthy prostate epithelial cells in preclinical experiments involving advanced mouse models of prostate cancer. The therapy not only killed cells at the primary tumor site, but also in distant metastases by "bystander" antitumor activity driven by the secreted MDA-7/IL-24 protein.
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Inovio Initiates Clinical Trial With DNA Immunotherapies to Prevent and Treat Ebola

Inovio Pharmaceuticals, Inc. announced today that the company has initiated a phase I trial to evaluate safety, tolerability and immune responses of Inovio's DNA immunotherapy for Ebola. In previously published preclinical testing, Inovio's DNA-based Ebola immunotherapy protected 100% of immunized animals from death and sickness after being exposed to a lethal dose of the Ebola virus. This is the first step in the Inovio-led consortium selected by the U.S. Defense Advanced Research Projects Agency (DARPA) to take a multi-faceted approach to develop products to both prevent and treat Ebola infection.
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Advanced Viral Gene Therapy Eradicates Prostate Cancer in Preclinical Experiments

Even with the best available treatments, the median survival of patients with metastatic, hormone-refractory prostate cancer is only two to three years. Driven by the need for more effective therapies for these patients, researchers at VCU Massey Cancer Center and the VCU Institute of Molecular Medicine (VIMM) have developed a unique approach that uses microscopic gas bubbles to deliver directly to the cancer a viral gene therapy in combination with an experimental drug that targets a specific gene driving the cancer's growth. Recently published in the journal Oncotarget, this new study is the most recent in a long line of studies led by Paul B. Fisher, M.Ph., Ph.D., investigating the use of viral gene therapy to treat a variety of cancers. The treatment strategy uses a novel "cancer terminator virus" (CTV), which replicates exclusively in cancer cells delivering the cancer-specific, toxic cytokine gene mda-7/IL-24 directly to the tumor. The researchers added an experimental drug known as BI-97D6, which targets MCL-1 and other members of the Bcl-2 gene family that protect cancer cells from therapeutic agents, resulting in enhanced prostate cancer cell death while sparing healthy prostate epithelial cells in preclinical experiments involving advanced mouse models of prostate cancer. The therapy not only killed cells at the primary tumor site, but also in distant metastases by "bystander" antitumor activity driven by the secreted MDA-7/IL-24 protein.
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Woodlands Based DNA Plasmid Manufacturer Receives $45M Grant for Ebola Vaccine

A breakthrough in infectious disease control, specifically Ebola, is evolving, and it’s taking place right here in The Woodlands. A $45 million dollar DARPA (Defense Advanced Research Projects Agency) grant was awarded to Inovio Pharmaceuticals, Inc., that has enabled the company to produce the vaccine at the facility of its manufacturing partner, VGXI in The Woodlands, in an effort to expedite the development for prevention and treatment of Ebola. Due to the global concerns and immediacy of need, an aggressive timeline has been set for the development of the Ebola vaccine. A Phase 1 clinical trial will begin within a couple of weeks to test the vaccine.
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Immunotherapy Protects Against CMV After Cord Blood Transplant

A novel adoptive immunotherapy offers a way to protect against potentially lethal cytomegalovirus (CMV) infection after hematopoietic stem cell transplant from umbilical cord blood or CMV-seronegative adult donors, hint the results of a phase 1 pilot study. In a first, researchers were able to create CMV-specific T-cells from CMV-naive T-cells derived from umbilical cord blood and adult CMV-seronegative donors. They infused the novel T-cells into three consecutive children who had undergone cord blood transplant and were at high risk for CMV infection. The procedure appeared safe and potentially protective. There was no infusion-related toxicity or graft-vs-host disease, and the children remain free of CMV more than 2 years after cord blood transplant, the researchers report. One patient experienced a reactivation of both CMV, which resolved with treatment; the other two patients have shown no signs of infection.
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Gene Therapy Company Acquires Rights to Eye Injector Technology

Spark Therapeutics Inc. has acquired the exclusive rights to a technology designed to deliver gene therapies to the back of the eye. The Philadelphia gene therapy company is initially focused on developing new treatments for inherited diseases that cause blindness. Specific financial terms of the deal between Spark (NASDQ: ONCE) and Clearside Biomedical, the Georgia-based developer of the microinjector technology, were not disclosed. The companies plan to explore using the technology to deliver viral vectors — the vehicles containing gene therapy treatment — to the choriod [the pigmented vascular layer of the eyeball] and the retina.
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Study Finds that Gene Therapy can Clip out Genetic Material Associated with Heart Failure

Gene therapy can clip out genetic material linked to heart failure and replace it with the normal gene in human cardiac cells, according to a study led by researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai. The study is published in the April 29 edition of Nature Communications. "Genetic mutations, the small random, changes in the genetic code making up genes, are major culprits in the weakening heart muscle seen in patients with heart failure," says Roger J. Hajjar, MD, Director of the Cardiovascular Research Center of Mount Sinai Heart at Icahn School of Medicine at Mount Sinai. "Our study results reveal that specific forms of heart failure can now be targeted by a new kind of gene therapy that acts as 'molecular scissors' cutting out the mutation and inserting the normal genetic code in its place."
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A CRISPR Antiviral Tool

Emory scientists have adapted an antiviral enzyme from bacteria called Cas9 into an instrument for inhibiting hepatitis C virus in human cells. The results were published Monday April 27, 2015 in Proceedings of the National Academy of Sciences. Cas9 is part of the SRISPR genetic defense system in bacteria, which scientists have been harnessing to edit DNA in animals, plants, and even human cells. In this case, Emory researchers are using Cas9 to put a clamp on RNA, which hepatitis C virus uses for its genetic material, rather than change cells' DNA.
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