Novartis/gene therapy: corporate DNA

The control premium for AveXis is hefty, but should be given the benefit of the doubt
Source: FT.com - Drugs and Healthcare - Category: Pharmaceuticals Source Type: news

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Small, EarlyView.
Source: Small - Category: Nanotechnology Authors: Source Type: research
AbstractGNE myopathy, previously known as hereditary inclusion body myopathy (HIBM), or Nonaka myopathy, is a rare autosomal recessive muscle disease characterized by progressive skeletal muscle atrophy. It has an estimated prevalence of 1 to 9:1,000,000. GNE myopathy is caused by mutations in theGNE gene which encodes the rate-limiting enzyme of sialic acid biosynthesis. The pathophysiology of the disease is not entirely understood, but hyposialylation of muscle glycans is thought to play an essential role. The typical presentation is bilateral foot drop caused by weakness of the anterior tibialis muscles with onset in ea...
Source: Neurotherapeutics - Category: Neurology Source Type: research
Publication date: 16 October 2018Source: Cell Reports, Volume 25, Issue 3Author(s): Suddhasil Mookherjee, Holly Yu Chen, Kevin Isgrig, Wenhan Yu, Suja Hiriyanna, Rivka Levron, Tiansen Li, Peter Colosi, Wade Chien, Anand Swaroop, Zhijian WuSummaryMutations in CEP290 cause ciliogenesis defects, leading to diverse clinical phenotypes, including Leber congenital amaurosis (LCA). Gene therapy for CEP290-associated diseases is hindered by the 7.4 kb CEP290 coding sequence, which is difficult to deliver in vivo. The multi-domain structure of the CEP290 protein suggests that a specific CEP290 domain may complement diseas...
Source: Cell Reports - Category: Cytology Source Type: research
iger MW Abstract Gene therapy for inherited eye diseases requires local viral vector delivery by intraocular injection. Since large animal models are lacking for most of these diseases, genetically modified mouse models are commonly used in preclinical proof-of-concept studies. However, because of the relatively small mouse eye, adverse effects of the subretinal delivery procedure itself may interfere with the therapeutic outcome. The method described here aims to provide the details relevant to perform a transscleral pars plana virus-mediated gene transfer to achieve an optimized therapeutic effect in the small m...
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
s S Abstract Gene therapy holds promise for treating previously untreatable retinal disorders. The most promising approaches use gene transfer vectors derived from adeno-associated virus (AAV) to supplement a gene function in the affected cell type. One example is gene therapy for achromatopsia which affects daylight vision. In this case, recombinant AAV (rAAV) vectors are being developed to specifically target cone photoreceptors. Development of rAAV vectors could be facilitated by the use of in vitro models. In this chapter we provide a protocol which utilizes mouse 661W cells, an in vitro model of cone photorec...
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Sarepta Therapeutics Inc. plans to open the first phase of its Gene Therapy Center of Excellence in Columbus by spring and expand throughout 2019 – eventually 85,000 square feet and 100 high-paying jobs. The CEO thinks Columbus could be for gene therapy what Cambridge is for biotech.
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
Publication date: January–February 2019Source: Diabetes &Metabolic Syndrome: Clinical Research &Reviews, Volume 13, Issue 1Author(s): Sin Yee Tan, Joyce Ling Mei Wong, Yan Jinn Sim, Su Sie Wong, Safa Abdelgadir Mohamed Elhassan, Sean Hong Tan, Grace Pei Ling Lim, Nicole Wuen Rong Tay, Naveenya Chetty Annan, Subrat Kumar Bhattamisra, Mayuren CandasamyAbstractType 1 and type 2 diabetes mellitus is a serious and lifelong condition commonly characterised by abnormally elevated blood glucose levels due to a failure in insulin production or a decrease in insulin sensitivity and function. Over the years, prevalence ...
Source: Diabetes and Metabolic Syndrome: Clinical Research and Reviews - Category: Endocrinology Source Type: research
Until a month ago, 4-year-old Caspian Soto had to use a headlamp and cane to navigate the world around him. He couldn ’t see the stars in the sky, or anything much in dim light. The Portland boy was born with a rare genetic mutation that causes Leber’s congenital amaurosis, a condition that made his vision dark and blurry. That all changed for Caspian when he underwent a new gene therapy treatment at the OHSU Casey Eye Institute called Luxturna. Doctors injected a modified virus into Caspian’s…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
(Massachusetts Institute of Technology) MIT biological engineers have devised a way to regulate the expression of messenger RNA once it gets into cells, giving them more precise control over gene therapy treatments for cancer and other diseases.
Source: EurekAlert! - Cancer - Category: Cancer & Oncology Source Type: news
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