A Novel OPA3 Mutation Revealed by Exome Sequencing

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A novel OPA3 mutation revealed by exome sequencing: an example of reverse phenotyping.

IMPORTANCE We sought to unravel the genetic cause in a consanguineous Pakistani family with a complex neurological phenotype. OBSERVATIONS Neurological and ophthalmological examination, including videotaping and fundoscopy, and genetic investigations, including homozygosity mapping and exome sequencing, were performed at the University of the Punjab and the University of Lübeck. Participants ...

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Charcot-Marie-Tooth disease (CMT) is the most common hereditary neuropathy of the peripheral nervous system with a wide range of severity and age of onset. CMT patients share similar phenotypes which make it often impossible to identify the disease types based on clinical presentation and electrophysiological studies alone. In recent years, novel genetic diagnostic approaches such as whole exom...

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ژورنال

عنوان ژورنال: JAMA Neurology

سال: 2013

ISSN: 2168-6149

DOI: 10.1001/jamaneurol.2013.1174