Gene therapy restores immunity in infants with rare immunodeficiency disease

NIH scientists and funding contributed to development of experimental treatment A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells do not develop or function normally. Eight infants with the disorder, called X-linked severe combined

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The Landscape of Cellular and Gene Therapy Products: Cost, Approvals, and Discontinuations

The past 10 years witnessed a significant increase in the approval of cellular and gene therapy products worldwide. The US Food and Drug Administration (FDA) approved 3 gene therapy products within the last 4 months of 2017. The objective of this study was to examine the approval characteristics, discontinuations and cost of all cellular and

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Two Patients Treated with CRISPRed Cells in Immunotherapy Trial

Researchers have infused cells edited using CRISPR-Cas9 into two patients in a trial conducted at the University of Pennsylvania, NPR reports today (April 16). A university spokesperson confirmed in an emailed statement to The Scientist that the trial is underway and that two patients, one with multiple myeloma and one with sarcoma, have been treated so far. The study is

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Study: Half of people on statins don’t hit healthy cholesterol levels

Cardiovascular disease (CVD) remains the in the United States. And one of the most effective preventive measures to lower the risk of a CVD event are statins — a class of cholesterol-lowering medications. It’s well established that statins save lives, but a recent study finds that even after two years, half of all people prescribed statins don’t achieve healthy cholesterol levels.

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Industry updates from the field of stem cell research and regenerative medicine in February 2019

Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from nonacademic institutions in February 2019. Business development Collaborations, partnerships & alliances Collaboration agreement: Pluristem & NASA Pluristem Therapeutics (Israel; www.pluristem.com) has collaborating with the NASA’s Ames Research Center to evaluate the potential of Pluristem’s PLX cell

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