Gene Therapy Treats Sickle Cell Disease in Teenager Using Lentiviral Vector

Gene therapy that delivered an antisickling variant of hemoglobin in an autologous hematopoietic stem cell (HSC) transplant has ameliorated symptoms of severe sickle cell disease (SCD) in a 15-year-old boy, according to a report published in the March 2 issue of the New England Journal of Medicine. SCD results from a point mutation at amino acid position 6 in the beta globin gene that causes polymerization of the protein and sickling of the erythrocytes that contain it, under low-oxygen conditions, obstructing microcirculation and damaging organs. Hemoglobin A (HbA) is the normal subunit; HbS is the one altered by the sickle cell mutation
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Bluebird Bio Announces Publication of Case Study on First Patient with Severe Sickle Cell Disease Treated with Gene Therapy in The New England Journal of Medicine

Bluebird bio, Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, today announced the publication in the New England Journal of Medicine of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and a brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016.
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DNA May Offer Rapid Road to Zika Vaccine

Last August, scientists injected a potential vaccine for Zika virus into a human being — just 3½ months after they had decided exactly what molecular recipe to use. In the world of vaccine development, 3½ months from design to injection is “warp speed,” says vaccine researcher Nelson Michael of the Walter Reed Army Institute of Research in Silver Spring, Md. Clinical trials can take years and epidemics can burn out before vaccines make it to doctors’ shelves. Even vaccine creation is typically sluggish. But in this case, the vaccine is a bit of DNA, which means scientists can get moving fast. Unlike some traditional methods, DNA vaccines don’t use dead or weakened viruses. Instead, they rely on a snippet of genetic material. This “naked” DNA carries, for example, the blueprints for Zika proteins. It’s just a long sequence of DNA blocks.
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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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