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Latest Adeno-associated virus Stories

2011-09-06 00:00:00

AMSTERDAM, September 6, 2011 /PRNewswire/ -- Gains US Patent Protection for Proprietary AAV Intellectual Property Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has entered into an exclusive license agreement with the US National Institutes of Health (NIH) for use of adeno-associated virus serotype 5 (AAV5)-based gene therapy vectors for liver and brain...

2011-08-25 00:00:00

AMSTERDAM, The Netherlands, August 25, 2011 /PRNewswire/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today reported its results for the first half year of 2011. Highlights - Glybera(R): - Data showing Glybera produces significant reduction in risk of pancreatitis in LPLD patients presented at European Atherosclerosis Society Meeting - CHMP does not...

2011-08-15 22:33:32

In a kind of molecular gymnastics, scientists at the University of North Carolina at Chapel Hill School of Medicine have devised a gene therapy cocktail that has the potential to treat some inherited diseases associated with "misfolded" proteins. Like strings of beads attached end-to-end on a chain, a given sequence of a protein's amino acids usually folds into a characteristic, three-dimensional structure. When "misfolded," a mutant protein's natural biological role may be compromised,...

2011-06-26 20:00:00

--Children's Hospital of Philadelphia Study Advances New Strategy for Gene Therapy-- PHILADELPHIA, June 26, 2011 /PRNewswire-USNewswire/ -- Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice. This is the first time that genome editing, which precisely targets and repairs a genetic defect, has been done in a living animal and achieved clinically...

2011-06-24 00:00:00

AMSTERDAM, June 24, 2011 /PRNewswire/ -- - Dossier Re-Examination Process Initiated Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera(R) (alipogene tiparvovec) as a potential therapy for Lipoprotein Lipase Deficiency ("LPLD"). Following a recent meeting with the European Medicines Agency's (EMA) Committee for Medicinal...

2011-05-26 07:05:00

FORT LEE, N.J., May 26, 2011 /PRNewswire/ -- Neurologix, Inc. (OTCBB: NRGX) announced the presentation today of efficacy results through one year of follow-up in patients treated as part of the Company's successful Phase 2 clinical trial for its novel, investigational gene therapy NLX-P101 for the treatment of Parkinson's disease (PD). Improvements in the Unified Parkinson's Disease Rating Scale (UPDRS) at 12 months for the randomized, double-blind, sham surgery-controlled trial of 45...

2011-05-23 00:00:00

AMSTERDAM, May 23, 2011 /PRNewswire-FirstCall/ -- Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today the results from studies exploring the role of adeno-associated viral (AAV) vectors for the efficient delivery of short and micro RNA to inhibit disease by RNA interference. Data related to the future development of an RNAi gene therapy for hypercholesterolemia and Huntington's disease were presented at the recent American Society...

2011-05-18 16:34:38

Many gene therapy strategies designed to deliver a normal copy of a gene to cells carrying a disease-causing genetic mutation rely on a modified virus to transfer the gene product into affected tissues. One technology platform that is well suited for in vivo delivery of genes is based on adeno-associated viruses (AAV). As these novel therapies move closer to commercialization, so do the methods for large-scale production and efficient delivery of AAV vectors, which are documented in a series...

2011-01-05 22:55:33

Described in Human Gene Therapy A new, simplified method for producing large amounts of viral vector cassettes capable of shuttling genes into host cells will help advance the promising field of gene therapy as applications move into large animal studies and human clinical trials. The novel adeno-associated virus (AAV) production method is described in an article published Instant Online ahead of publication in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc....

2010-12-07 01:52:45

Vector-gene combination developed at St. Jude Children's Research Hospital and UCL is central to latest clinical trial of gene therapy as a possible tool to provide long-term relief from disabling bleeds Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inherited bleeding disorder. This trial was...