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Tag: Gene Therapy

Thorough CRISPR
In a study published in Genome Biology, researchers explain how they are working to make CRISPR less prone to large, accidental deletions of genetic material [1]. A previously unknown large problem While this seems counterintuitive, previous technology only allowed for the ready identification of deletions below 100 base pairs. New technology has allowed for larger...
George Church Interview
Professor of Genetics at Harvard Medical School and one of the most prominent geroscientists, George Church works on gene therapies that can potentially reverse age-related diseases. We had the opportunity to interview this prolific researcher and entrepreneur, who is involved in dozens of startups, on topics ranging from the current state of gene therapy to...
Neuropathy
Intellia has released clinical data for NTLA-2001, a gene therapy for hereditary transthyretin amyloidosis. What is transthyretin amyloidosis? Transthyretin (ATTR) amyloidosis is the buildup of misfolded transthyretin, a protein generated by the liver. Normally, this protein is partially responsible for the transport of thyroid hormones, hence its name; however, transthyretin is vulnerable to mutations and...
Neurons
Scientists have successfully improved learning and memory, as well as neuronal morphology, in Alzheimer’s-prone mice with the help of a gene therapy that upregulates a vital protein [1]. Alzheimer’s disease (AD) has been a notoriously hard problem to crack, despite major R&D investment. AD has long been associated with the accumulation of amyloid-beta (Aß) plaques...
Image of scissors cutting DNA to represent gene editing with CRISPR
The body can be home to many latent viruses, which can lie dormant for years only to be activated much later under certain conditions. Now, researchers may have the solution to one such persistent virus: herpes simplex virus 1, which is commonly known as oral herpes. Latent viruses put strain on the immune system A...
The Journal Club returns August 25th at 13:00 EDT live on our Facebook page where Dr. Oliver Medvedik will be taking a look at the new study by Dr. Maria Blasco from the CNIO, which shows that telomerase therapy can address fibrosis in normally aged mice and not just mouse models of the disease. Short/dysfunctional...