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alipogene tiparvovec

National Institutes of Health

Papers overview

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2018
2018
One-year results are reported of the first lipoprotein lipase deficiency (LPLD) patient treated with alipogene tiparvovec, which… Expand
Highly Cited
2014
Highly Cited
2014
Cellular immune responses to adeno-associated viral (AAV) vectors used for gene therapy have been linked to attenuated transgene… Expand
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Review
2014
Review
2014
There has been great interest over the past two decades in developing gene therapies (GTs) to treat a variety of diseases… Expand
Highly Cited
2013
Highly Cited
2013
We describe the 2-year follow-up of an open-label trial (CT-AMT-011–01) of AAV1-LPLS447X gene therapy for lipoprotein lipase (LPL… Expand
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2013
2013
Homozygous lipoprotein lipase (LPL) deficiency is an ultra-orphan disease associated with increased rates of pancreatitis… Expand
2013
2013
Alipogene tiparvovec is the first adeno-associated virus (AAV)-mediated gene therapy to be approved for the treatment of a… Expand
Highly Cited
2012
Highly Cited
2012
BACKGROUND Lipoprotein lipase-deficient (LPLD) individuals display marked chylomicronemia and hypertriglyceridemia associated… Expand
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Highly Cited
2012
Highly Cited
2012
A recent news article discussed the progress in gene therapy, a field that has had a number of major setbacks (Gene therapies… Expand
2009
2009
Amsterdam Molecular Therapeutics BV is developing alipogene tiparvovec (Glybera, AMT-011, AAV1-LPLS447X), a Ser(447)X variant of… Expand
2009
2009
Introduction: Lipoprotein lipase deficiency (LPLD) is the most common cause of familial chylomicronemia and severe… Expand