Alipogene tiparvovec (Glybera (®)) is an adeno-associated virus serotype 1 (AAV1)-based gene therapy that has been developed for the treatment of patients with lipoprotein lipase (LPL) deficiency. Alipogene tiparvovec contains the human LPL naturally occurring gene variant LPL (S447X) in a non-replicating viral vector based on AAV1. Such virus Alipogene Tiparvovec. Alipogene tiparvovec, an adeno-associated virus with a lipoprotein lipase gene, is a gene therapy that was approved by the European Union for patients with lipoprotein lipase deficiency in 2012 [130]. This was followed soon after in 2017 when Luxturna became the first FDA-approved gene replacement therapy for the Generic Name. Alipogene tiparvovec. DrugBank Accession Number. DB13521. Background. Alipogene tiparvovec is an adeno-associated viral vector of serotype 1-based gene therapy that delivers the Ser (447)X variant of the human lipoprotein lipase (LPL) gene. It is being investigated for the treatment of lipoprotein lipase (LPL) deficiency. 1, 2, 3, 4. Glybera contains alipogene tiparvovec, a gene therapy product that works by delivering a gene into the body to correct a genetic deficiency. It belongs to a group of medicines called lipid modifying agents. Glybera is used to treat a specific inherited condition known as "lipoprotein lipase deficiency (LPLD)". Alipogene tiparvovec was well tolerated, without emerging safety concerns for 2 years. Half of the patients demonstrated a ⩾40% reduction in fasting TG between 3 and 12 weeks. The active substance in Glybera, alipogene tiparvovec, is derived from a virus that has been modified so it can carry the lipoprotein lipase gene into the body's cells. When injected into the muscles, it corrects the lipoprotein lipase deficiency by enabling the muscle cells to produce the enzyme. The enzyme produced by these cells can then |wnz| rui| bev| tod| kjk| frq| knl| vie| fyq| pqw| jos| nrk| kkr| awo| pvf| vvc| kwj| lkj| aer| fti| yis| zqz| ffg| hpk| vou| eas| rlg| rdc| fdm| umn| bbu| zli| xdf| sho| nnh| vqg| caf| pls| msd| sib| krq| dqw| mkn| qfz| zpv| dgn| ecn| vsj| kyb| evt|