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Tag: Gene Therapy

Gene pill
In a study printed in PNAS, researchers have shown that telomerase reverse transcriptase (TERT) can be given to cells in living mice through a viral vector, taking the idea of life-extending gene therapies from science fiction to reality. Why a cytomegalovirus? The human cytomegalovirus (CMV) is widely known as an endemic virus that, while usually...
Aging DNA
In a new study, mice transduced with a longevity-associated variant (LAV) of the BPIFB4 gene showed less immunosenescence and healthier vasculature [1]. The importance of having good genes Longevity-associated alleles prove that aging does not affect everyone equally. On average, centenarians and supercentenarians are more protected from age-related diseases than other people, and this protection...
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...

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