Clinical development on the frontier: gene therapy for duchenne muscular dystrophy

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Source: Expert Opinion on Biological Therapy - Category: Drugs & Pharmacology Authors: Source Type: research

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Abstract Rapid progress in knowledge of the organization of the dog genome has facilitated the identification of the mutations responsible for numerous monogenic diseases, which usually present a breed-specific distribution. The majority of these diseases have clinical and molecular counterparts in humans. The affected dogs have thus become valuable models for preclinical studies of gene therapy for problems such as eye diseases, immunodeficiency, lysosomal storage diseases, hemophilia, and muscular dystrophy. Successful gene therapies in dogs have significantly contributed to decisions to run clinical trials for ...
Source: J Appl Genet - Category: Genetics & Stem Cells Authors: Tags: J Appl Genet Source Type: research
This article reviews recent developments in the field of antisense and gene therapies for hereditary muscle diseases. PMID: 32076754 [PubMed - as supplied by publisher]
Source: Der Nervenarzt - Category: Neurology Authors: Tags: Nervenarzt Source Type: research
Abstract The limb-girdle muscular dystrophies (LGMD) are genetically heterogeneous disorders, responsible for muscle wasting and severe form of dystrophies. Despite the critical developments in the insight and information of pathomechanisms of limb-girdle muscular dystrophy, any definitive treatments do not exist, and current strategies are only based on the improvement of the signs of disorder and to enhance the life quality without resolving an underlying cause. There is a crucial relationship between pharmacological therapy and different consequences; therefore, other treatment strategies will be required. New ...
Source: Current Gene Therapy - Category: Genetics & Stem Cells Authors: Tags: Curr Gene Ther Source Type: research
Abstract The gene therapy approach aiming at curing various human diseases began to develop as a technology from early eighties of the last century. To date the delivery of therapeutic genes are mainly mediated by virus-based, predominantly, non-integrated virus vectors. These gene delivery approaches have several fundamental limitations on the way of efficient deployment in clinical gene therapy. A totally different approach was suggested about 20 years ago when episomal non-integrative artificial chromosome-based vectors featuring large size inserts (even native gene loci) advanced to the stage. Since then numer...
Source: Experimental Cell Research - Category: Cytology Authors: Tags: Exp Cell Res Source Type: research
Andrew is the “And” in Andelyn Biosciences. He was 3 in 2001 when he was diagnosed with Duchenne muscular dystrophy, the most common and among the most aggressive forms of the inherited condition. Patients usually don’t reach 20.
Source: bizjournals.com Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.“With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant,&rdqu...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news
(Technical University of Munich (TUM)) Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy. Researchers at Technical University of Munich (TUM), Ludwig Maximilian University of Munich (LMU) and the German Research Center for Environmental Health (Helmholtz Zentrum M ü nchen) have developed a gene therapy that may provide permanent relief for those suffering from DMD.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
In this study, we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD.
Source: Genes and Diseases - Category: Genetics & Stem Cells Source Type: research
In this study, we briefly explained the molecular genetics of DMD and a historical overview of DMD gene therapy. We in particular focused on CRISPR/Cas9-mediated therapeutic approaches that used to treat DMD.
Source: Genes and Diseases - Category: Genetics & Stem Cells Source Type: research
A gene therapy invented at Nationwide Children's Hospital just landed a licensing deal worth as much as $2.8 billion for a Boston biotech, and now the company's Columbus, Ohio office will play a big role in getting the treatment to the market. Sarepta Therapeutics Inc. has licensed the commercial rights outside of the United States to the Swiss pharmaceutical giant Roche for $1.1 billion in cash and stock up front. Sarepta also can earn as much as $1.7 billion in milestone payments, plus royalties.…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news
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