Glowing Mice Suggest New Gene Therapy Technique

A collaboration between chemists and gene therapy experts produced a new way of inserting the code for modified proteins into the cells of mice. If successful in humans, the technique could be useful for vaccines or cancer therapies. This research was made possible through coordination between the chemists and experts in imaging molecules in live animals, who rarely work together directly. With this partnership, the synthesis, characterization and testing of compounds could take as little as a week.
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Broad Institute Wins Big Battle Over CRISPR Gene-Editing Patent

The U.S. patent office has delivered a potentially lucrative victory to bioengineer Feng Zhang of the Broad Institute in Massachusetts, regarding patents for an extraordinarily useful gene-editing tool. CRISPR, a technology that's already worth billions of dollars, is shaping up to play a big role in medicine and medical research because it can edit DNA with unprecedented accuracy. But exactly who has the right to profit from the technology has been up for debate. Wednesday the U.S. Patent and Trademark Office said patents issued to the Broad Institute in 2014, and then challenged by the University of California, Berkeley, are in fact valid.
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RNA Vaccine for Zika Shows Promise

In the last year, a diverse suite of anti-Zika vaccines has rapidly advanced through preclinical development and into human trials. A Phase 1 trial testing one approach, which relies on DNA to encode vaccine components, has already yielded encouraging results. Now, another nucleic acid, messenger RNA (mRNA) is joining the Zika-vaccine toolbox. According to a study published in Nature today (February 2), a single shot of a vaccine containing Zika virus mRNA encapsulated in a lipid nanoparticle induced protective immune responses in both mice and Rhesus macaques. “It’s a novel vaccine platform. It has not been explored in great detail in humans yet, but it will have more clinical experience going forward,” said Dan Barouch, who directs the Center for Virology and Vaccine Research at Harvard Medical School and was not involved in the present study.
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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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