Gene Therapy May Be Cure for Rare Blood Disorder

There are an estimated 288,000 cases of beta-thalassemia across the world, making it one of the most common genetic diseases, according to an editorial accompanying the study.
Source: WebMD Health - Category: Consumer Health News Source Type: news

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miR-140-5p inhibits the proliferation and enhances the efficacy of doxorubicin to breast cancer stem cells by targeting Wnt1, Published online: 22 July 2018; doi:10.1038/s41417-018-0035-0miR-140-5p inhibits the proliferation and enhances the efficacy of doxorubicin to breast cancer stem cells by targeting Wnt1
Source: Cancer Gene Therapy - Category: Cancer & Oncology Authors: Source Type: research
Over at the Life Extension Advocacy Foundation (LEAF), an argument is made that we shouldn't be overly concerned about the current unwillingness of the FDA and similar regulatory bodies to recognize the treatment of aging and its causes as a valid indication. What is an indication? The outcome of the present overly burdensome regulatory process is formal approval of the use of a specific medical technology for a specific defined condition or set of symptoms. That condition or set of symptoms is known as an indication. Aging is not currently in the list of recognized conditions. The argument made by LEAF is that there are d...
Source: Fight Aging! - Category: Research Authors: Tags: Politics and Legislation Source Type: blogs
The long-term success of prosthetic vascular grafts for revascularization in peripheral arterial disease is limited by the development of anastomotic neointimal hyperplasia. We have constructed a bioactive prosthetic graft material by coating polyethylene terephthalate (PET) with an antithrombogenic cryogel polymer layer capable of delivering biologic agents in vitro. Our goal was to evaluate our hybrid prosthetic graft material in vivo using a rabbit carotid interposition bypass model.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Tags: Abstract from the 2018 Eastern Vascular Society Annual Meeting Source Type: research
Publication date: Available online 19 July 2018Source: Nano TodayAuthor(s): Lotte M.P. Vermeulen, Toon Brans, Stefaan C. De Smedt, Katrien Remaut, Kevin BraeckmansAbstractA plethora of biological barriers, intended to defend tissues and cells against external influences, stand in the way of efficient nucleic acid delivery by non-viral nanoplexes. Even when nanoplexes successfully evade extracellular barriers and reach their target cell, many intracellular barriers remain to be conquered. These include overcoming the plasma membrane, evading endosomal compartmentalization, and in some cases crossing the nuclear envelope. At...
Source: Nano Today - Category: Nanotechnology Source Type: research
Category for table of contents – immunotherapy / gene therapy
Source: Experimental Hematology - Category: Hematology Authors: Tags: Review Source Type: research
New Jersey-based drug developer PTC Therapeutics Inc. announced Thursday that it is purchasing Agilis Biotherapeutics Inc., a Lynnfield gene therapy biotech, for $200 million in cash and stock. PTC (Nasdaq: PTCT) said it would pay $50 million in cash and $150 million in PTC stock to acquire Agilis. The privately held biotech is developing gene therapies for rare and often fatal nervous system diseases, including a disorder called AADC Deficiency that inhibits motor control and the abili ty to swallow…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
Downregulation of hypoxia‑inducible factor-1α inhibits growth, invasion, and angiogenesis of human salivary adenoid cystic carcinoma cells under hypoxia. Oncol Rep. 2018 Jul 12;: Authors: Xiao C, Pan Y, Zeng X, Wang L, Li Z, Yan S, Wang H Abstract Surgical and medical treatments usually fail to completely remove the primary lesions of salivary adenoid cystic carcinoma (SACC), resulting in local recurrence due to its strong infiltration, hematogenous metastasis and other unique biological behaviors. Targeted gene therapy, including hypoxia‑inducible factor‑1α (HIF‑1α) therapy, ...
Source: Oncology Reports - Category: Cancer & Oncology Tags: Oncol Rep Source Type: research
Authors: Xin G, Chen R, Zhang X Abstract The aim of the present study was to explore the candidate genes, chemicals and mechanisms of congenital obstructive nephropathy (CON). The gene expression profiles of GSE48041, including 24 kidney tissue samples from megabladder (mgb‑/‑) mouse were downloaded from the Gene Expression Omnibus database. Samples were divided into 4 groups: Control, mild, moderate and severe. Differentially expressed genes (DEGs), protein‑protein interaction network, Kyoto Encyclopedia of Genes and Genomes pathways and transcription factor (TF)‑target gene analyses were perform...
Source: Molecular Medicine Reports - Category: Molecular Biology Tags: Mol Med Rep Source Type: research
Identification of a novel idiopathic congenital nystagmus‑causing missense mutation, p.G296C, in the FRMD7 gene. Mol Med Rep. 2018 Jul 09;: Authors: Xiu Y, Yao Y, Yang T, Pan M, Yang H, Fang W, Gu F, Zhao J, Zhu Y Abstract Exploring the genetic basis for idiopathic congenital nystagmus is critical for improving our understanding of its molecular pathogenesis. In the present study, direct sequencing using gene specific primers was performed in order to identify the causative mutations in two brothers from a Chinese family who had been diagnosed with idiopathic congenital nystagmus. A comprehensive oph...
Source: Molecular Medicine Reports - Category: Molecular Biology Tags: Mol Med Rep Source Type: research
Publication date: Available online 17 July 2018Source: Regulatory Toxicology and PharmacologyAuthor(s): Frank R. Brennan, Laura Andrews, Antonio R. Arulanandam, Jorg Blumel, Jim Fikes, Christine Grimaldi, Janice Lansita, Lise I. Loberg, Tim MacLachlan, Mark Milton, Suezanne Parker, Jay Tibbitts, Jayanthi Wolf, Krishna P. AllamneniAbstractNonclinical safety testing of biopharmaceuticals can present significant challenges to human risk assessment with these innovative and often complex drugs. Emerging topics in this field were discussed recently at the 2016 Annual US BioSafe General Membership meeting. The presentations and ...
Source: Regulatory Toxicology and Pharmacology - Category: Toxicology Source Type: research
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