AveXis to Advance Gene Therapy Program Initiated by the Rett Syndrome...

The Rett Syndrome Research Trust (RSRT) is excited to announce that AveXis, Inc., has committed to advance a gene therapy candidate for Rett Syndrome into human clinical trials.(PRWeb June 08, 2017)Read the full story at http://www.prweb.com/releases/2017/06/prweb14408379.htm
Source: PRWeb: Medical Pharmaceuticals - Category: Pharmaceuticals Source Type: news

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Publication date: Available online 12 August 2020Source: Brain StimulationAuthor(s): Katherine S. Adcock, Collin Chandler, Elizabeth P. Buell, Bleyda R. Solorzano, Kristofer W. Loerwald, Michael S. Borland, Crystal T. Engineer
Source: Brain Stimulation - Category: Neurology Source Type: research
Abstract Stem cells are characterized by self-renewal and by their ability to differentiate into cells of various organs. With massive progress in 2D and 3D cell culture techniques, in vitro generation of various types of such organoids from patient-derived stem cells is now possible. As in vitro differentiation protocols are usually made to resemble human developmental processes, organogenesis of patient-derived stem cells can provide key information regarding a range of developmental diseases. Human stem cell-based in vitro modeling as opposed to using animal models can particularly benefit the evaluation of neu...
Source: Archives of Pharmacal Research - Category: Drugs & Pharmacology Authors: Tags: Arch Pharm Res Source Type: research
cute;a F, Lanuza E, Santos M, Agustín-Pavón C Abstract Deficits in arginine vasopressin (AVP) and oxytocin (OT), two neuropeptides closely implicated in the modulation of social behaviours, have been reported in some early developmental disorders and autism spectrum disorders. Mutations in the X-linked methyl-CpG-binding protein 2 (MECP2) gene are associated to Rett syndrome and other neuropsychiatric conditions. Thus, we first analysed AVP and OT expression in the brain of Mecp2-mutant mice by immunohistochemistry. Our results revealed no significant differences in these systems in young adult Mecp2...
Source: Brain Structure and Function - Category: Neuroscience Authors: Tags: Brain Struct Funct Source Type: research
AbstractDeficits in arginine vasopressin (AVP) and oxytocin (OT), two neuropeptides closely implicated in the modulation of social behaviours, have been reported in some early developmental disorders and autism spectrum disorders. Mutations in the X-linked methyl-CpG-binding protein 2 (MECP2) gene are associated to Rett syndrome and other neuropsychiatric conditions. Thus, we first analysed AVP and OT expression in the brain ofMecp2-mutant mice by immunohistochemistry. Our results revealed no significant differences in these systems in young adultMecp2-heterozygous females, as compared to WT littermates. By contrast, we fo...
Source: Anatomy and Embryology - Category: Anatomy Source Type: research
AON-platform with focus on therapies nearing phase I trials for forms of Spinocerebellar Ataxia and Huntington Disease Other early discovery stage RNA editing platform focuses on RETT syndrome Funding led by LSP, co-led by Kurma Partners, supported b... Biopharmaceuticals, Neurology, Venture Capital Vico Therapeutics, RNA modulating therapies
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
(Salk Institute) In research that aims to illuminate the causes of human developmental disorders, Salk scientists have generated 168 new maps of chemical marks on strands of DNA -- called methylation -- in developing mice. The data can help narrow down regions of the human genome that play roles in diseases such as schizophrenia and Rett Syndrome.
Source: EurekAlert! - Biology - Category: Biology Source Type: news
Abstract Difficulties with communication have a profound impact on the lives of individuals with Rett syndrome and their caregivers. Globally, many families report difficulty accessing appropriate and timely information and services from professionals with expertise in augmentative and alternative communication (AAC) as it pertains to Rett syndrome. To address this need, international consensus-based guidelines for managing the communication of individuals with Rett syndrome were developed by combining available evidence and lived experience with expert opinion. A two-phase Delphi survey was built on statements an...
Source: Augmentative and Alternative Communication - Category: Speech-Language Pathology Authors: Tags: Augment Altern Commun Source Type: research
Publication date: August 2020Source: The Lancet Neurology, Volume 19, Issue 8Author(s): Jan Egebjerg, Richard G M Morris
Source: The Lancet Neurology - Category: Neurology Source Type: research
Publication date: August 2020Source: The Lancet Neurology, Volume 19, Issue 8Author(s): Alexander J Sandweiss, Vicky L Brandt, Huda Y Zoghbi
Source: The Lancet Neurology - Category: Neurology Source Type: research
To investigate walking ability in Japanese patients with Rett syndrome (RTT).
Source: Brain and Development - Category: Neurology Authors: Tags: Original article Source Type: research
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