Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic.
نویسندگان
چکیده
Tom Adriaenssens, MD, PhD; Michael Joner, MD; Thea C. Godschalk, MSc; Nikesh Malik, MD; Fernando Alfonso, MD, PhD; Erion Xhepa, MD; Dries De Cock, MD; Kenichi Komukai, MD; Tomohisa Tada, MD; Javier Cuesta, MD; Vasile Sirbu, MD; Laurent J. Feldman, MD, PhD; Franz-Josef Neumann, MD; Alison H. Goodall, PhD; Ton Heestermans, MD; Ian Buysschaert, MD, PhD; Ota Hlinomaz, MD; Ann Belmans, MSc; Walter Desmet, MD; Jurrien M. ten Berg, MD, PhD; Anthony H. Gershlick, MD; Steffen Massberg, MD; Adnan Kastrati, MD; Giulio Guagliumi, MD; Robert A. Byrne, MB, BCh, PhD; on behalf of the Prevention of Late Stent Thrombosis by an Interdisciplinary Global European Effort (PRESTIGE) Investigators
منابع مشابه
P164: Adeno-Associated Viral Vectors in Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
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Background Duchenne Muscular Dystrophy (DMD) is a deadly X-linked recessive disorder. This genetic disorder affects 1 among 3,500-5,000 males in the world. The majority of the patients are male, due to the type of inheritance. It affects most of the skeletal, the respiratory, and cardiac muscles, causing these vital organs to contract and eventually mortality.<br...
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Duchenne muscular dystrophy (DMD) is the most common childhood neuromuscular disorder. It is caused by mutations in the DMD gene that disrupt the open reading frame (ORF) preventing the production of functional dystrophin protein. The loss of dystrophin ultimately leads to the degeneration of muscle fibres, progressive weakness and premature death. Antisense oligonucleotides (AOs) targeted to s...
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Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease caused by frame shifting and nonsense mutations in the dystrophin gene. Through skipping of an (additional) exon from the pre-mRNA, the reading frame can be restored. This can be achieved with antisense oligonucleotides (AONs), which induce exon skipping by binding to splice sites or splice enhancer sites. The resulting protei...
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ورودعنوان ژورنال:
- Circulation
دوره 136 11 شماره
صفحات -
تاریخ انتشار 2017