Gene-therapy
Gene-therapy
Latest Posts
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Gene therapy using lipid based nanoparticles
Lipid nanoparticles (SLNs and NLCs) are regarded as highly promising systems for delivering nucleic acids in gene therapy. Literature review by researchers at PharmaNanoGene describes these systems and their main advantages in gene therapy, such as their capacity to protect…
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Making T-Cell receptor gene therapy safer by domain swapping
The human body produces T cells to recognize and fight disease. Each T cell has a unique T cell receptor (or TCR) on its surface that surveils small fragments of proteins presented by other cells. Upon detecting…
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Gene therapy with Alipogene Tiparvovec (Glybera) for Lipoprotein Lipase Deficiency shows promise
Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the severity and frequency of pancreatitis. No cases of…
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Researchers study foamy retrovirus as a safe gene therapy vector
A Washington State University researcher has developed a way to reduce the development of cancer cells that are an infrequent but dangerous byproduct of gene therapy. Grant Trobridge, an associate professor of pharmaceutical sciences, has altered the…
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Research finds that gene therapy could be used in early Alzheimers disease in animal studies
Researchers have prevented the development of Alzheimer’s disease in mice by using a virus to deliver a specific gene into the brain.The early-stage findings, by scientists from Imperial College London, open avenues for potential new treatments for…
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Biosafety studies of Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis I shows promise for human trials
Extensive biosafety studies of hematopoietic stem cell (HSC) gene therapy, intended to replace a protein that patients with the inherited disease mucopolysaccaridosis I (MPS I) cannot produce, support clinical testing of the stem cell-based gene addition approach…
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Gene therapy for Cystic fibrosis updates
Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF). Working with CF pigs, the researchers, based in the…
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