Design of a promising gene therapy method to treat Wilson's disease

(Universidad de Navarra) Spanish scientists at the University of Navarra have designed VTX801, a promising gene therapy method to treat Wilson's disease. They expected to begin the first clinical trials on patients in 2018. The Massachusetts Eye and Ear Infirmary, associated with Harvard University, has participated in its development. This Spanish research project has attracted leading international investors in biotechnology by forming Vivet Therapeutics, an emerging company which has raised € 37.5 million in its Series A financing round.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news

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Human Gene Therapy,Volume 30, Issue 12, Page 1494-1504, December 2019.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Authors: Woimant F, Djebrani-Oussedik N, Collet C, Girardot N, Poujois A Abstract In brief, the classic form of Wilson's disease (WD) is an autosomal-recessive condition with hepatic, neurologic, psychiatric and systemic manifestations. However, the diagnosis should not be excluded because of a family history consistent with autosomal-dominant transmission. The latest next-generation sequencing (NGS) studies have demonstrated a gap between phenotype and genetic prevalences, and also suggest that WD may still be underdiagnosed. In a majority of WD patients, early recognition and appropriate treatment can result in r...
Source: Revue Neurologique - Category: Neurology Tags: Rev Neurol (Paris) Source Type: research
Publication date: Available online 4 April 2018Source: Clinics and Research in Hepatology and GastroenterologyAuthor(s): Aurélia Poujois, France WoimantSummaryWilson's disease (WD) is characterised by a deleterious accumulation of copper in the liver and brain. It is one of those rare genetic disorders that benefits from effective and lifelong treatments that have dramatically transformed the prognosis of the disease. In Europe, its clinical prevalence is estimated at between 1.2 and 2/100,000 but the genetic prevalence is higher, at around 1/7000. Incomplete penetrance of the gene or the presence of modifier genes ...
Source: Clinics and Research in Hepatology and Gastroenterology - Category: Gastroenterology Source Type: research
CONCLUSION: The aim of this review is to provide a comprehensive clinical profile and to review the current available therapies. We overview the physiopathology, neurological features and diagnostic approach of the common recessive ataxias. The emphasis is also put on potential drugs currently or soon-to-be in clinical trials. Promising gene therapies raise the possibility of treating specifically Friedreich's ataxia, Ataxia-telangiectasia, Wilson's disease and Niemann-Pick disease in the next few years. PMID: 29676235 [PubMed - as supplied by publisher]
Source: CNS and Neurological Disorders Drug Targets - Category: Drugs & Pharmacology Authors: Tags: CNS Neurol Disord Drug Targets Source Type: research
Publication date: Available online 4 April 2018 Source:Clinics and Research in Hepatology and Gastroenterology Author(s): Aurélia Poujois, France Woimant Wilson's disease (WD) is characterised by a deleterious accumulation of copper in the liver and brain. It is one of those rare genetic disorders that benefits from effective and lifelong treatments that have dramatically transformed the prognosis of the disease. In Europe, its clinical prevalence is estimated at between 1.2 and 2/100,000 but the genetic prevalence is higher, at around 1/7000. Incomplete penetrance of the gene or the presence of modifier genes may ...
Source: Clinics and Research in Hepatology and Gastroenterology - Category: Gastroenterology Source Type: research
Abstract Wilson's disease (WD) is an autosomal recessive disorder of copper metabolism, caused by mutations in the ATP7B gene. A clear demand for novel WD treatment strategies has emerged. Although therapies using zinc salts and copper chelators can effectively cure WD, these drugs exhibit limitations in a substantial pool of WD patients who develop intolerance and/or severe side effects. Several lines of research have indicated intriguing potential for novel strategies and targets for development of new therapies. Here, we review these new approaches, which comprise correction of ATP7B mutants and discovery of ne...
Source: World Journal of Gastroenterology : WJG - Category: Gastroenterology Authors: Tags: World J Gastroenterol Source Type: research
Publication date: 2017 Source:Handbook of Clinical Neurology, Volume 142 Author(s): Christian Rupp, Wolfgang Stremmel, Karl-Heinz Weiss Wilson disease is an autosomal-recessive copper overload disorder causing hepatic and neurologic symptoms. Commonly used medical therapy shows satisfactory results with regard to hepatic disease but only limited effects in neurologically affected patients. In recent years several new therapy options have been developed, showing promising results that might improve the management of Wilson disease in the near future. Optimization of treatment regimens depending on biochemical response patt...
Source: Handbook of Clinical Neurology - Category: Neurology Source Type: research
We describe requirements for cell therapy in animal models with several standardized methods for studies to test or refine cell therapy strategies in WD. PMID: 27830552 [PubMed - in process]
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Current treatments for Wilson Disease (WD) are based on lifelong copper chelating drugs, which may cause side effects and do not restore normal copper metabolism. In this work, Murillo et al. assessed the efficacy of gene therapy by transducing the liver of the Atp7b−/− WD mouse model with an adenovirus encoding the human ATP7B cDNA. They observed a dose-dependent therapeutic effect of the gene therapy approach manifested by the reduction of serum transaminases and urinary copper excretion, normalization of serum holoceruloplasmin, and restoration of physiological biliary copper excretion in response to copper overload.
Source: Journal of Hepatology - Category: Gastroenterology Authors: Tags: Editorial Desk Source Type: research
Wilson disease (WD) was a once progressive and uniformly fatal inherited disorder of copper metabolism. Medical therapy to arrest progression or prevent complications of WD was developed in the 1950’s with the introduction of parenterally administered BAL[1], and over the next two decades by oral therapy with d-penicillamine trientine, and zinc [2]. Effective therapy for WD requires life-long administration of daily medication. At least 30-50% of patients on medication for their WD have periods of non-adherence, some suffering liver failure, others developing potentially irreversible neurologic or psychiatric symptoms.
Source: Journal of Hepatology - Category: Gastroenterology Authors: Source Type: research
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