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10.1093/hmg/ddv442 http://scihub22266oqcxt.onion/10.1093/hmg/ddv442 C4802374!4802374!26503961     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=26503961&cmd=llinks): Failed to open stream: HTTP request failed! 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Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington s disease and spinocerebellar ataxia |pdf=|usr=}}{{26503961}} gab.com Text Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington s disease and spinocerebellar ataxia JO: Hum Mol Genet http://www.kidney.de/pget.php?&tw=1&pmid=26503961 #FOAvip RNA-targeting approaches are emerging as viable therapeutics that offer an alternative method to modulate traditionally ?undrugable? targets. In the case of dominantly inherited neurodegenerative diseases, gene suppression strategies can target the underlying cause of these intractable disorders. Polyglutamine diseases are caused by CAG expansions in discrete genes, making them ideal candidates for gene suppression therapies. Here, we discuss the current state of gene suppression approaches for Huntington's disease and the spinocerebellar ataxias, including the use of antisense oligonucleotides, short-interfering RNAs, as well as viral vector-mediated delivery of short hairpin RNAs and artificial microRNAs. We focus on lessons learned from preclinical studies investigating gene suppression therapies for these disorders, particularly in rodent models of disease and in non-human primates. In animal models, recent advances in gene suppression technologies have not only prevented disease progression in a number of cases, but have also reversed existing disease, providing evidence that reducing the expression of disease-causing genes may be of benefit in symptomatic patients. Both allele- and non-allele-specific approaches to gene suppression have made great strides over the past decade, showing efficacy and safety in both small and large animal models. Advances in delivery techniques allow for broad and durable suppression of target genes, have been validated in non-human primates and in some cases, are currently being evaluated in human patients. Finally, we discuss the challenges of developing and delivering gene suppression constructs into the CNS and recent advances of potential therapeutics into the clinic. Twit Text FOAVip Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington s disease and spinocerebellar ataxia ????Hum Mol Genet http://www.kidney.de/pget.php?&tw=1&pmid=26503961 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=26503961 #FOAvip English Wikipedia <ref>{{Cite pmid|26503961}}</ref> Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington s disease and spinocerebellar ataxia #MMPMID26503961 Keiser MS; Kordasiewicz HB; McBride JL Hum Mol Genet 2016[Apr]; 25 (R1): R53-64 PMID26503961show ga RNA-targeting approaches are emerging as viable therapeutics that offer an alternative method to modulate traditionally ?undrugable? targets. In the case of dominantly inherited neurodegenerative diseases, gene suppression strategies can target the underlying cause of these intractable disorders. Polyglutamine diseases are caused by CAG expansions in discrete genes, making them ideal candidates for gene suppression therapies. Here, we discuss the current state of gene suppression approaches for Huntington's disease and the spinocerebellar ataxias, including the use of antisense oligonucleotides, short-interfering RNAs, as well as viral vector-mediated delivery of short hairpin RNAs and artificial microRNAs. We focus on lessons learned from preclinical studies investigating gene suppression therapies for these disorders, particularly in rodent models of disease and in non-human primates. In animal models, recent advances in gene suppression technologies have not only prevented disease progression in a number of cases, but have also reversed existing disease, providing evidence that reducing the expression of disease-causing genes may be of benefit in symptomatic patients. Both allele- and non-allele-specific approaches to gene suppression have made great strides over the past decade, showing efficacy and safety in both small and large animal models. Advances in delivery techniques allow for broad and durable suppression of target genes, have been validated in non-human primates and in some cases, are currently being evaluated in human patients. Finally, we discuss the challenges of developing and delivering gene suppression constructs into the CNS and recent advances of potential therapeutics into the clinic. äGene suppression strategies for dominantly inherited neurodegenerative(epmc).pdf DeepDyve Pubget Overpricing