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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… 
Review
2016
Review
2016
Alipogene tiparvovec (Glybera) is a gene therapy product approved in Europe under the "exceptional circumstances" pathway as a… 
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… 
Review
2014
Review
2014
Alipogene tiparvovec (Glybera®; AMT-011, AAV1-LPLS447X) is an adeno-associated virus serotype 1-based gene therapy for adult… 
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… 
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… 
2013
2013
Homozygous lipoprotein lipase (LPL) deficiency is an ultra-orphan disease associated with increased rates of pancreatitis… 
Highly Cited
2012
Highly Cited
2012
BACKGROUND Lipoprotein lipase-deficient (LPLD) individuals display marked chylomicronemia and hypertriglyceridemia associated… 
2009
2009
Amsterdam Molecular Therapeutics BV is developing alipogene tiparvovec (Glybera, AMT-011, AAV1-LPLS447X), a Ser(447)X variant of…