CLINICAL TRIALS 101
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Landmark CRISPR Trial Shows Promise Against Deadly Disease
Source: https://www.nature.com/articles/d41586-021-01776-4
Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease. The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver …
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CRISPR Injected Into the Blood Treats a Genetic Disease for First Time
Source: https://www.science.org/content/article/crispr-injected-blood-treats-genetic-disease-first-time
The gene editor CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing an enormous hurdle: getting the molecular scissors into the body and having it slice DNA in the tissues where it’s needed. Now, in a medical first, researchers have injected a CRISPR …
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Gene Editing Therapy Paves Way for Revolution in Treatment of Genetic Disorders
People with hereditary transthyretin (ATTR) amyloidosis have a mutation in the transthyretin (TTR) gene, which means they produce an abnormal protein that gradually builds up in the heart and nerves. Symptoms include numbness in the hands and feet, loss of control of the bowel and bladder and immobility. The condition gets progressively worse and is …
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