Forough Taheri, Eskandar Taghizadeh, Mohammad J.R. Pour, Mohammadali Raeisi, Pedram G. Renani, Azam Rastgar-Moghadam and Seyed M.G. Hayat* Pages 386 - 394 ( 9 )
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 approaches, such as gene replacement, gene transfer, exon skipping, siRNA knockdown, and anti-myostatin therapy, which can target specific cellular or molecular mechanism of LGMD, could be a promising avenue for the treatment. Recently, genome engineering strategies with a focus on molecular tools such as CRISPR-Cas9 are used to different types of neuromuscular disorders and show the highest potential for clinical translation of these therapies. Thus, recent advancements and challenges in the field will be reviewed in this paper.
Adeno-associated virus, exon skipping, gene therapy, limb-girdle muscular dystrophy, LGMD, gene editing.
Shahrekord Branch, Islamic Azad University, Shahrekord, Cellular and Molecular Research Center, Yasuj University of Medical Sciences, Yasuj, Department of Biology, Faculty of Sciences, Mashhad-Branch, Islamic Azad University, Mashhad, Department of Neurology, Pastor Hospital, Bam University of Medical Sciences, Bam, Shahrekord Branch, Islamic Azad University, Shahrekord, Department of Genetics, Tehran Medical Sciences Branch, Islamic Azad University, Tehran, Department of Medical Genetics, School of Medicine, Shahid Sadoughi University of Medical Sciences, Yazd