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Category Archives: Genetic Therapy

AveXis SMA gene therapy advances toward pivotal trial – FierceBiotech

AveXis has posted top-line data from a phase 1 trial of its gene therapy against spinal muscular atrophy (SMA). All 15 patients were event-free at 13.6 months of age, a significant improvement over the natural history of the disease. The phase 1 gave three babies with the genetic muscle weakness disease a low dose of a gene therapy designed to provide them with a functioning SMN gene Continue reading



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AveXis SMA gene therapy advances toward pivotal trial - FierceBiotech

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This Breakthrough Approach In Gene Therapy Can Potentially Revolutionize Our Fight Against Cancer and HIV – Wall Street Pit

This Breakthrough Approach In Gene Therapy Can Potentially Revolutionize Our Fight Against Cancer and HIV Developed by scientists at Fred Hutchinson Cancer Research Center in Seattle and led by Dr. Jennifer Adair, the novel treatment is being referred to as gene therapy in a box. It is designed to deliver potential HIV and cancer treatments to the poorest of nations, the ones without sufficient resources for state-of-the-art facilities and infrastructure Continue reading



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This Breakthrough Approach In Gene Therapy Can Potentially Revolutionize Our Fight Against Cancer and HIV - Wall Street Pit

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Treating sickle cell disease with gene therapy – Jamaica Observer

After nearly two years of debate about its possible benefits and risks, the gene editing technique is now here to stay. An article in the December 27, 2015 edition of the Sunday Observer told of the first recorded use of the inexpensive CASPR-Cas9 gene editing technology to cut and splice out bad genes and replace them with healthy genes. Continue reading



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Treating sickle cell disease with gene therapy - Jamaica Observer

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Sickle cell cure? Patient in complete remission following gene therapy – Genetic Literacy Project

A number of recent headlines imply a recent case study just publishedproves that gene therapy has cured sickle cell diseasea genetic disorder that incurs tremendous pain, suffering and diminished life expectancy. Due to such limited progress in management of this condition, this team of researcherstook samples from the bone marrow of a patient with severe diseaseUsing a lentiviral vector, they transferred an anti-sickling gene into the patients stem cellswhich get put back into the patient in the hope they will multiply and replace the cells made with the defective gene Continue reading



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Sickle cell cure? Patient in complete remission following gene therapy - Genetic Literacy Project

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Tocagen seeks $86M IPO to fund cancer gene therapy trial … – FierceBiotech

Tocagen has filed to raise up to $86 million in an IPO to take its breakthrough-designated brain cancer gene therapy combination through the first part of a phase 2/3 trial. Continue reading



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Tocagen seeks $86M IPO to fund cancer gene therapy trial ... - FierceBiotech

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Amid advances in gene therapy, ‘bubble baby’ in SF gains hope – San Francisco Chronicle

JaCeon Golden has only ever known the inside of hospitals. But the treatment hes receiving may have implications far beyond his as-yet isolated life. Round-faced and big-eyed, with a perpetual pout that belies his sunny nature, he looks as healthy as any other 5-month-old Continue reading



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Amid advances in gene therapy, 'bubble baby' in SF gains hope - San Francisco Chronicle

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