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Category Archives: Genetic Therapy

Pioneering stem cell gene therapy cures infants with bubble baby disease – UCLA Newsroom

FINDINGS UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated. In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured Continue reading

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French Gene Therapy Biotech Gets Ready for Phase II/III – Labiotech.eu (blog)

Lysogene has crunched the numbers on its observational study for Sanfillipo A now the company can move into the final phase for its Sanfilippo A therapy. Lysogene is a French biotech specializing in gene therapy of rare CNS diseases. Continue reading

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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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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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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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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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