The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...
By the time Enid Katz was two years old, she had already suffered through several bouts of bronchitis and was always hungry, ...
The TP53 gene is a gene that is mutated in many cancers. It is the most common gene mutation found in cancer cells. A tumor-suppressor gene, TP53 codes for a protein that inhibits the development and ...
for the treatment of cystic fibrosis in patients with at least one F508del mutation or another treatment-responsive mutation in the CFTR gene. The fixed-dose triple-combination therapy ...
BOSTON, January 12, 2025--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the ...
The first human trial of insulin-producing cells that have been gene-edited to evade immune attack is a success so far. The cells have survived and produced insulin for a month after being ...
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This complex process, known as gene expression, determines which proteins we make and whether we make them in the correct amounts, crucial work given that proteins play a role in virtually every a ...
A baby boy born with a debilitating genetic disease will likely no longer require a liver transplant to stabilize his condition, thanks to a first … ...
Summary: Researchers have developed an AI model that accurately predicts gene activity in any human cell, providing insights into cellular functions and disease mechanisms. Trained on data from over 1 ...
The same is now possible in gene editing. Rather than employ separate tools for editing genes and regulating their expression, these distinct goals can now be combined into a single tool that can ...
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