Scientists Restore Partial Hearing and Balance in Deaf Mice Using New Gene Therapy

Using a novel form of gene therapy, scientists from Harvard Medical School and the Massachusetts General Hospital have managed to restore partial hearing and balance in mice born with a genetic condition that affects both. The new model overcomes a long-standing barrier to accessing hair cells, the delicate sensors in the inner ear that capture sound and head movement and convert them to neural signals for hearing and balance. These cells have been notoriously difficult to treat with previous gene-delivery techniques. The team's findings, published in the February issue of Molecular Therapy, show that the treatment leads to notable gains in hearing and allows mice that would normally be completely deaf to hear the equivalent of a loud conversation. The approach also improved the animals' sense of balance.
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Gene Therapy for Pompe Disease Effective in Mice, Poised for Human Trials

After decades investigating a rare, life-threatening condition that cripples the muscles, Duke Health researchers have developed a gene therapy they hope could enhance or even replace the only FDA-approved treatment currently available to patients. The gene therapy, demonstrated in mice, is described in a new study published online in the journal Molecular Therapy - Methods & Clinical Development. The therapy uses a modified virus to deliver a gene to the liver where it produces GAA, an enzyme missing in people with Pompe disease.
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Gene Therapy is Ready to Become Hereditary

Gene therapy is about ready to be the next big thing. The prospect of treating diseases by tinkering with DNA has a long history of both promise and frustration. Steady progress means 2017 should be the year the technology finally hits the U.S. market. The problem may be figuring out how to pay for cures. Two or more gene therapies soon could be approved in the United States. GlaxoSmithKline's treatment for a rare and deadly immune disease probably will get the green light from regulators on the strength of the data. Smaller biotech Spark Therapeutics also will seek signoff for a therapy to restore vision in an inherited form of progressive blindness.
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Cost-Effective Cas9 – Higher Concentrations Bring Results

CRISPR/Cas9 is a vital part of our research at the University of Minnesota and the Cas9 recombinant protein, used at high concentration, has allowed for highly efficient modification of T-cells. By introducing a Cas9 nuclease guide RNA complex (RNP), we target a specific spot in the genome, where the nuclease cuts the DNA. The DNA break is repaired in one of two ways: homologous recombination, which is high-fidelity, or non-homologous endjoining (NHEJ), which is more error-prone.
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Program to Study ALS Gene Therapy, Genome Editing Launched at Penn

A new program at the University of Pennsylvania will pursue ways to use gene therapy and genome editing to treat amyotrophic lateral sclerosis (ALS). The Program of Excellence for Motor Neuron Disease, launched by researchers at the Orphan Disease Center (ODC) in the university’s Perelman School of Medicine, will receive its initial funding from philanthropic sources. “I am convinced that it is time to make a serious effort to treat ALS using gene therapy,” James Wilson, MD, PhD, director of the ODC and its affiliated gene therapy program, said in a press release. “To do so, we will leverage the exciting clinical results that have been achieved in gene therapy for spinal muscular atrophy using our vectors, as well as the robust infrastructure in gene therapy translational research we have at the Orphan Disease Center and Gene Therapy Program at Penn.”
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Immunotherapy, Gene Therapy Combination Shows Promise Against Glioblastoma

New research out of the University of Michigan supports combining two approaches to fight back against gliomas: attacking the tumor with gene therapy while enhancing the immune system's ability to fight it, too. The paper was recently published in Molecular Therapy, the official journal of the American Society of Gene & Cell Therapy. "We hope the implementation of our gene therapy strategy for gliomas, used in combination with immune checkpoint blockade, will eventually provide successful treatment for patients with this devastating brain cancer," says Maria Castro, Ph.D., co-senior author and U-M professor of neurosurgery and cell and developmental biology.
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Novel DNA Vaccine for Peanut Allergy Gets Fast Tracked

Astellas and Immunomic announced that the Food and Drug Administration (FDA) has granted Fast Track designation to its candidate ASP0892 for the mitigation of severe hypersensitivity reactions due to peanut allergy. ASP0892 is a novel DNA vaccine based on the investigational LAMP-Vax platform. Unlike conventional DNA vaccines, the LAMP-Vax vaccine includes a short DNA sequence encoding the Lysosomal Associated Membrane Protein (LAMP). This method may allow the vaccine to utilize the body's natural biochemistry to produce a more complete immune response. This response can include antibody production, cytokine release, and critical immunological memory.
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MeiraGTx Expands Neurodegenerative Gene Therapy Pipeline in Collaboration with Weill Cornell Medicine

MeiraGTx, a New York and UK based gene therapy company, announces the expansion of its Neurodegenerative Disease gene therapy pipeline. Research into the causes of neurodegenerative disease has converged upon the central idea that misfolded proteins play a critical role in damaging neuronal function and the ultimate progression to neuronal cell death. The new program, a collaboration with Dr. Greg Petsko of Weill Cornell Medicine, targets neurodegenerative disease by altering neuronal protein processing. Leveraging seminal work from Dr. Petsko, the Arthur J. Mahon Professor of Neuroscience and Director of its Helen and Robert Appel Alzheimer's Disease Research Institute in the Feil Family Brain and Mind Research Institute at Weill Cornell Medicine, and his colleagues, the new program targets one of the master regulators of protein trafficking, known as retromer. Dr. Petsko and colleagues have developed genetic strategies to reprogram retromer, impacting protein trafficking, processing and aggregation. Using a gene therapy approach, the team aims to modulate retromer function to treat several inherited and age-related neurodegenerative diseases including Alzheimer's and Parkinson's disease.
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Key Insight About Mitochondrial Replacement Therapy

A new discovery may unlock the answer to a vexing scientific question: How to conduct mitochondrial replacement therapy, a new gene-therapy technique, in such a way that safely prevents the transmission of harmful mitochondrial gene mutations from mothers to their children. A study to be published Nov. 30 in the journal Nature suggests that clinicians select egg donors whose mitochondrial DNA (mtDNA) is compatible to the mother's ancestral mitochondria. Similar groups of mitochondrial DNA are known as haplotypes, each of which represents major branching points on the human genetic family tree. "This research suggests that we're going to have the greatest success rates for producing an embryo free of disease-causing genetic mutations by making sure we are using the right combination of haplotypes," said senior author Shoukhrat Mitalipov, Ph.D., who directs the Center for Embryonic Cell and Gene Therapy at OHSU.
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New Research Shows How Synthetic DNA Vaccine Approach Protects Against Zika Virus Infection

As the global spread of the Zika virus continues, efforts are underway to halt the disease's transmission. While no licensed therapies or vaccines to protect against the Zika virus are currently available, new research published in the journal npj Vaccines demonstrates how a synthetic DNA vaccine approach successfully protected against infection, brain damage and death caused by the mosquito-borne Zika virus in vivo. In this preclinical study, 100 percent of the animal models were protected from Zika after vaccination followed by a challenge with the Zika virus. In addition, they were protected from degeneration in the cerebral cortex and hippocampal areas of the brain, while the other cohort showed degeneration of the brain after Zika infection.
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