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Genetic base editing treats sickle cell disease in mice

Broad Institute of MIT and Harvard
Sickle cell disease (SCD) is the most common deadly genetic disorder, affecting more than 300,000 newborns worldwide each year. It leads to chronic pain, organ failure, and early death in patients. A team led by researchers at the Broad Institute of MIT and Harvard and St. Jude Children’s Research Hospital has now demonstrated a base editing approach that efficiently corrects the mutation underlying SCD in patient blood stem cells and in mice. This gene editing treatment rescued the disease symptoms in animal models, enabling the long-lasting production of healthy blood cells.
The root of SCD is two mutated copies of the hemoglobin gene, HBB, which cause red blood cells to transform from a circular disc into a sickle shape setting off a chain of events leading to organ damage, recurrent pain, and early mortality. In this study, the researchers used a molecular technology called base editing to directly convert a single letter of patho ....

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Pairwise Raises $90 Million to Bring New Varieties of Fruits and Vegetables to Market

Press release content from Business Wire. The AP news staff was not involved in its creation.
Pairwise Raises $90 Million to Bring New Varieties of Fruits and Vegetables to Market
February 3, 2021 GMT
DURHAM, N.C. (BUSINESS WIRE) Feb 3, 2021
Pairwise announced the closing of its $90 million Series B funding round, signaling a new frontier in healthy, sustainable food innovation. Funding for the round was led by Pontifax Global Food and Agriculture Technology Fund (Pontifax AgTech), a pioneering growth capital investor in food and agriculture technology, and existing investor Deerfield Management Company (Deerfield), an investment firm dedicated to advancing healthcare through investment, information and philanthropy. Also joining the Series B round are new investor Temasek, an investment company headquartered in Singapore, and existing investor Leaps by Bayer, which invests in paradigm-shifting advances in the life sciences sector, including agriculture and health ....

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Breakthrough Study Supports Genetic Editing as a Potential Treatment for Rare Rapid-Aging Disease Progeria

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PEABODY, Mass., Jan. 6, 2021 /PRNewswire/
The Progeria Research Foundation (PRF) announced today the publication of a breakthrough preclinical mouse study in the journal
Nature, demonstrating the substantial effect of genetic editing in directly reversing the mutation that causes Hutchinson-Gilford Progeria Syndrome (HGPS, or Progeria). Scientists at Broad Institute, the National Institutes of Health (NIH), PRF and others contributed to this ground-breaking study, indicating a critical new pathway toward correcting the root cause of Progeria.
Progeria is an ultra-rare, fatal, pediatric rapid-aging disease, affecting approximately 400 children worldwide. Without the FDA-approved treatment Zokinvy (lonafarnib), children with Progeria die of heart disease at an average age of 14.5 years, due to premature atherosclerosis resulting in heart attacks. Zokinvy increases average lifespan by 2.5 years and improves some symptoms of Progeria, but it do ....

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