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Category Archives: Genetic Therapy

Girl, 6, is ‘thriving’ after experimental new gene-therapy DESTROYS her leukaemia – The Sun

A LITTLE girl who was diagnosed with deadly leuakemia has undergone a new therapy to destroy the disease. Erin Cross is now happy and thriving after having immunotherapy in the US, followed by a bone marrow transplant to wipe out the disease. [email protected] Her parents were devastated when doctors revealed last year that her acute lymphoblastic leukaemia had returned Continue reading

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Cutting-edge gene therapy provides hope for patients with inherited eye conditions – Miami Herald

Miami Herald Cutting-edge gene therapy provides hope for patients with inherited eye conditions Miami Herald The trial will evaluate a cutting-edge concept: gene therapy . While there are no gene therapy products currently approved for use in the U.S., researchers are experimenting to see if they can provide a solution to alleviate hereditary diseases Continue reading

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Prosper nonprofit brings gene therapy treatment closer to reality – Star Local Media

Over the past three years, parent-led efforts have collectively raised half a million dollars to support gene therapy research at Nationwide Childrens Hospital (Columbus, Ohio) to treat the ultra-rare disease Hunter Syndrome (also known as Mucopolysaccharidosis or MPS II). Continue reading

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A gene therapy pioneer moves from Biogen to Regenxbio, staying … – Endpoints News

Olivier Danos arrival as head of Biogens gene therapy group two-and-a-half years ago signaled an important shift in the big biotechs R&D focus. In swift order Biogen executed a partnership with the San Raffaele-Telethon Institute for Gene Therapy, inked a $1 billion-plus deal with AGTC and followed up with a $2 billion pact to work with James Wilson and Jean Bennett at the University of Pennsylvania Continue reading

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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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