Gene Therapy Offers Hope for Treatment of Sickle Cell Anaemia

Scientists are finalizing plans to use gene therapy to treat one of the world’s most widespread inherited diseases – sickle cell anaemia. The technique could begin trials next year, say researchers. About 300,000 babies are born globally with sickle cell disease. The condition causes red blood cells to deform, triggering anaemia, pain, organ failure, tissue damage, strokes and heart attacks. In the west, patients now live to their 40s thanks to the availability of blood transfusions and other treatments. But in Africa most still die in childhood. Sickle cell anaemia is triggered by a genetic fault that changes one of the dozens of amino acids that make up haemoglobin, the key constituent of the red blood cells that carry oxygen around our bodies. The mutated haemoglobin undergoes a change in shape and blocks veins.
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Gene Therapy for Cystic Fibrosis Lung Disease

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF). Working with CF pigs, the researchers, based in the UI Pappajohn Biomedical Institute (PBI), have shown that two different virus-based vectors can restore a working version of the cystic fibrosis transmembrane conductance regulator (CFTR) protein that is faulty in CF to the pigs' airway cells. Moreover, this gene replacement normalized important aspects of the lung biology and improved the ability of airway secretions to kill bacteria.
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Promising Gene Therapy For Sickle Cell Ready for Clinical Trial

A new engineered gene therapy virus, inserted into blood stem cells and then transplanted into mice with sickle cell disease, markedly reduced red blood cell damage according to the study “Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype,” published in the Journal of Clinical Investigation. A clinical gene therapy trial is expected in the coming year in which researchers will use a gene manipulated harmless virus to prevent the “sickling” of red blood cells. The new gene therapy is based on research going back to the 1980s which revealed that people with a milder form of sickle cell disease carried a fetal form of hemoglobin.
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Gene Therapy for LPLD Patients Linked to Lower Frequency and Severity of Pancreatitis

Up to 6 years after receiving a single treatment with the gene therapy product lipoprotein lipase (LPL), patients with the debilitating genetic disease LPL deficiency (LPLD) had about 50% fewer episodes of pancreatitis than before receiving the treatment. None of the study participants suffered severe pancreatitis following gene therapy and only one required admission to the intensive care unit for treatment of LPLD, which can be fatal, according to a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com/).
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Another DNA Vaccine for Zika Shows Promise

A preventive DNA vaccine encoding two Zika structural proteins protected Rhesus macaques from viral infection. The results, published today (September 22) in Science, are encouraging for organizers of the ongoing Phase 1 clinical trial testing one of the two vaccines examined in this nonhuman primate study. The new work suggests a minimal antibody level in the blood that is likely necessary for protection against Zika virus infection in in people. “This is a reassuring development and critical advance,” said Nelson Michael of the Walter Reed Army Institute of Research in Silver Spring, Maryland, whose team is testing a formalin-inactivated viral particle vaccine. Michael was not involved in the present study, but regularly communicates with its authors, sharing Zika-related data. “This [DNA vaccine], if proven safe and [that it] generates the type of immune response in humans seen here in animals, is on the path to potentially become the first public health tool to mitigate the Zika virus epidemic in the Americas,” he tol
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Smallest-Reported Artificial Virus Could Help Advance Gene Therapy

Gene therapy is a kind of experimental treatment that is designed to fix faulty genetic material and help a patient fight off or recover from a disease. Now scientists have engineered the smallest-reported virus-like shell that can self-assemble. It could someday carry potentially therapeutic DNA or RNA and transfer it to human cells. The report appears in the Journal of the American Chemical Society. The story of gene therapy is fraught with much hype and high-profile failures. But, hype and failures aside, it remains a promising route to treat a range of ailments, from rare genetic diseases to common conditions such as diabetes. Clinical trials to test various gene therapy treatments are underway. One possible approach is to copy the way viruses behave. When they infect people, viruses inject their genetic material into human cells. Artificial viruses have been engineered to mimic this step, but they tend to clump or are not uniform in size, which can hinder their effectiveness. Max Ryadnov and colleagues wanted to address these issues.
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Gene Therapy Technique May Help Prevent Cancer Metastasis

The spread of malignant cells around the body, known as metastasis, is the leading cause of mortality in women with breast cancer. Now, a new gene therapy technique being developed by researchers at MIT is showing promise as a way to prevent breast cancer tumors from metastasizing. The treatment, described in a paper published today in the journal Nature Communications, uses microRNAs — small noncoding RNA molecules that regulate gene expression — to control metastasis. The therapy could be used alongside chemotherapy to treat early-stage breast cancer tumors before they spread, according to Natalie Artzi, a principal research scientist at MIT’s Institute for Medical Engineering and Science (IMES) and an assistant professor of medicine at Brigham and Women’s Hospital, who led the research in collaboration with Noam Shomron, an assistant professor on the faculty of medicine at Tel-Aviv University in Is
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Novartis to Disband Cell & Gene Therapy Unit, 120 Jobs to go

Novartis is folding activities of its Cell and Gene Therapy unit into other business and research locations, eliminating 120 positions, the Swiss drugmaker said on Wednesday. The move intensifies a corporate makeover begun this year as it focuses on high-growth areas including cancer immunotherapy. Basel-based Novartis said the move will not derail its intentions to file CTL019, a chimeric antigen receptor T cell (CART) therapy, for treatment of young people with relapsed/refractory acute lymphoblastic leukemia with U.S. and European regulators in 2017. Nor will it disrupt a gene editing push that Novartis hopes will lead to new therapies for hard-to-treat diseases, it said.
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Novel Gene Therapy Drug for Huntington’s Disease

Data from Vybion on a novel treatment for Huntington's disease has been published in the Journal of Neurodegenerative diseases. The published study links the ability of Vybion's proprietary, novel Intrabody (INT41) blocking of cellular gene dysregulation to the delay of cognitive and motor function loss in the well-validated vR6/2 animal model. INT41 interferes with direct binding of toxic N-terminal huntingtin fragments to DNA, as well as their transport into the nucleus. The data support a direct gain of function of N-terminal huntingtin protein fragments that may lead to neuron dysfunction and brain atrophy as well as a novel therapeutic modality. The data also demonstrate that Vybion's platform may have broader application in generating Intrabody drug candidates to difficult-to-drug targets, including intracellular proteins in neurodegenerative disorders and other disease areas such as oncology. Further, the platform may be used to validate new targets of interest, particularly in intracellular signal transduction pathways, prior to generation of new therapeutic candidates.
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CRISPR-Cas9 Breaks Genes Better if you Disrupt DNA Repair

The popular gene-editing tool CRISPR-Cas9 competes with DNA repair, continually cutting what repair enzymes fix until the enzymes make a mistake, resulting in a broken gene. This led to a trick to improve cutting efficiency of the Cas9 protein. By dumping random bits of non-homologous DNA into the cell with Cas9, they disrupted the DNA repair process and boosted knockout efficiency up to five fold in human cell lines. University of California, Berkeley researchers have now found a way to boost the efficiency with which CRISPR-Cas9 cuts and disables genes up to fivefold, in most types of human cells, making it easier to create and study knockout cell lines and, potentially, disable a mutant gene as a form of human therapy.
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