Category Archives: Gene Therapy

Gene therapy trial for thalassemia at Royal Prince Alfred Hospital A world first gene therapy trial is currently underway at Royal Prince Alfred Hospital with the potential to cure patients living with thalassemia. Patient S... By: SydneyLHD … Continue reading →

Aarkstore -Gene Therapy Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape Gene Therapy Insight: Pipeline Assessment, Technology Trend, and Competitive Landscape provides the information across the gene therapy value chain covering gene therapy profiles core insights,... By: sangam Jain … Continue reading →

Switzerland: The Elixir of Life? Scientists COULD make you live to 120! Researchers from Bern University may have found gene therapy that can extend life by 60 percent. At the moment, the experiment is limited to fruit flies, who have just been given their third... By: RuptlyTV … Continue reading →

He added that one passage in the Bible, the Magnificat or the Song of Mary is so revolutionary he was surprised it was not banned as un-American during the McCarthy era. His comments came as he took part in a conference at the Trinity Wall Street church in New York discussing the idea of the common good. In an address on whether inequality matters, he argued that a message of basic equality can be traced through the Bible from the Garden of Eden to the New Testament accounts of the early church. He said that although wealth is, in some parts of the Bible, viewed as a blessing, in others it is linked to corruption but that overall there was no right to be rich. He added that some people had labelled passages in the Book of Acts, talking about wealth being shared between the early Christians, as communist but said this was untrue because the common ownership in the Bible was voluntary not obligatory. Looking ahead to the next 40 years, he said: In an era in which we will see the growth of technologies like Artificial Intelligence and gene therapies, economists like Lawrence Summers foresee growing inequalities between … Continue reading →

Minecraft: Attack of the B Team, Ep. 51 Gene therapy! In this episode of Attack of the B Team we put our advanced genetics knowledge to use, not good use, just use.... Really the best use for dna? https://www.fa... By: Dr. Trotter … Continue reading →

Methylmalonic Acidemia (MMA) Gene Therapy - Charles Venditti and Randy Chandler Dr. Charles Venditti and Dr. Randy Chandler discuss recent developments in gene therapy for methylmalonic acidemia (MMA) - a group of inherited disorders in which the body is unable to process... By: GenomeTV … Continue reading →

Washington, D.C.-basedgene therapy outfit Regenxbio just raised $30 million to bringits platforminto the clinic. The dollars will help Regenxbio generate clinical proof of concept data, as well as work toward in-licensing new programs. The companys also using the money to beef up its clinical and manufacturing processes, it said in a statement. Regenxbio has developed what it calls NAV Technology a form of adeno-associated viral gene therapy that treats lysosomal storage disorders and ocular disease. Its got drugs in the pipeline that treat Hurler syndrome, Hunter syndrome, wet age-related macular degeneration and X-linked retinitis pigmentosa. Heres how it works: In Hurler syndrome, for instance, children dont carry a gene that develops an enzyme called IDUA that breaks down complex sugars. These build up, and ultimately impairmental development, organ function, physical abilities and appearance. The NAV platform delivers a normal copy of the IDUA-producing gene which ultimately embeds itself into a patients DNA in a one-time doze so that patients can produce the enzyme. The research comes out of the University of Pennsylvania, and has been successful in vivo so its a matter of testing its efficacy in a real patient pool. The companys also out-licensing this technology for other indications, … Continue reading →

PHILADELPHIA, Penn., January 20, 2015- Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today it has initiated a Phase 1/2 clinical trial for the potential treatment of patients with choroideremia (CHM) utilizing its gene therapy product SPK-CHM. The Phase 1/2 trial is an open-label, dose-escalating trial designed to assess the safety and preliminary efficacy of sub-retinal administration of SPK-CHM. The Phase 1/2 trial will be conducted at The Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania, and has plans to enroll up to 10 patients afflicted with the CHM genetic mutation. "Spark's groundbreaking announcement today brings real hope of a treatment for blindness caused by choroideremia, and promises to pave the way for treatments of other retinal diseases impacting millions of people around the World," said Dr. Chris Moen, President of the Choroideremia Research Foundation (curechm.org), the leading advocacy and fundraising organization focused on finding a cure for CHM. "The Choroideremia Research Foundation is proud to have provided key pre-clinical funding to Jean Bennett, MD, PhD and her team atthe Perelman School of Medicine at the University of Pennsylvania,that has helped bring us to the gene therapy human clinical trials being announced … Continue reading →

Gene Therapy: Hope For The Blind? In medicine, doctors often talk about treatments, but rarely about cures. Now, gene therapy is offering the potential to actually cure certain diseases and an experimental breakthrough could... By: NewsChannel 5 … Continue reading →

Gene therapy is a promising strategy to correct hereditary disorders. The approach takes advantage of viral vectors to deliver a corrected version of the mutated gene. Adeno-associated virus (AAV) has many features that make it a favorable vector for gene therapy. In animal models, AAV-mediated gene delivery is generally regarded as safe and has demonstrated efficacy for some genetic diseases. However, a recent study reported an increase in liver cancer in mice after AAV gene therapy. A new publication in the Journal of Clinical Investigation reveals that AAV vector design influences the likelihood of developing cancer in the liver. Charles Venditti and colleagues at the National Institutes of Health looked for the development of hepatocellular carcinoma (HCC) in a large number of mice that had received AAV gene therapy. HCC was associated with the AAV vector integrating within a specific site in the genome and inducing expression of microRNAs and a retrotranposon. Moreover, AAV dose, the choice of enhancer/promoter, and timing of delivery all influenced the HCC incidence. The results of this study provide insight into features that should be considered when designing AAV vectors for gene therapy. ### TITLE: Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy … Continue reading →