Gene-therapy

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 the gene material against degradation, to facilitate cell and nucleus internalization and to boost the transfection process. View …

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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 severe pancreatitis and only one admission to the intensive care unit for an lipoprotein lipase deficiency related abdominal …

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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 way a virus carries a beneficial gene to its target cell. The modified viral vectors reduce the risk …

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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 in MPS I patients. Evidence derived from these studies not only indicates that the HSC gene therapy is …

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