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10.2147/TACG.S57682 http://scihub22266oqcxt.onion/10.2147/TACG.S57682 C4485851!4485851!26170711     free     free     free Deprecated: Implicit conversion from float 213.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Appl+Clin+Genet 2015 ; 8 (ä): 157-69 Nephropedia Template TP {{tp|p=26170711|t=2015. Recent advances in gene therapy for lysosomal storage disorders |pdf=|usr=}}{{26170711}} gab.com Text Recent advances in gene therapy for lysosomal storage disorders JO: Appl Clin Genet http://www.kidney.de/pget.php?&tw=1&pmid=26170711 #FOAvip Lysosomal storage disorders (LSDs) are a group of genetic diseases that result in metabolic derangements of the lysosome. Most LSDs are due to the genetic absence of a single catabolic enzyme, causing accumulation of the enzyme?s substrate within the lysosome. Over time, tissue-specific substrate accumulations result in a spectrum of symptoms and disabilities that vary by LSD. LSDs are promising targets for gene therapy because delivery of a single gene into a small percentage of the appropriate target cells may be sufficient to impact the clinical course of the disease. Recently, there have been several significant advancements in the potential for gene therapy of these disorders, including the first human trials. Future clinical trials will build upon these initial attempts, with an improved understanding of immune system responses to gene therapy, the obstacle that the blood?brain barrier poses for neuropathic LSDs, as well other biological barriers that, when overcome, may facilitate gene therapy for LSDs. In this manuscript, we will highlight the recent innovations in gene therapy for LSDs and discuss the clinical limitations that remain to be overcome, with the goal of fostering an understanding and further development of this important field. Twit Text FOAVip Recent advances in gene therapy for lysosomal storage disorders ????Appl Clin Genet http://www.kidney.de/pget.php?&tw=1&pmid=26170711 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=26170711 #FOAvip English Wikipedia <ref>{{Cite pmid|26170711}}</ref> Recent advances in gene therapy for lysosomal storage disorders #MMPMID26170711 Rastall DP; Amalfitano A Appl Clin Genet 2015[]; 8 (ä): 157-69 PMID26170711show ga Lysosomal storage disorders (LSDs) are a group of genetic diseases that result in metabolic derangements of the lysosome. Most LSDs are due to the genetic absence of a single catabolic enzyme, causing accumulation of the enzyme?s substrate within the lysosome. Over time, tissue-specific substrate accumulations result in a spectrum of symptoms and disabilities that vary by LSD. LSDs are promising targets for gene therapy because delivery of a single gene into a small percentage of the appropriate target cells may be sufficient to impact the clinical course of the disease. Recently, there have been several significant advancements in the potential for gene therapy of these disorders, including the first human trials. Future clinical trials will build upon these initial attempts, with an improved understanding of immune system responses to gene therapy, the obstacle that the blood?brain barrier poses for neuropathic LSDs, as well other biological barriers that, when overcome, may facilitate gene therapy for LSDs. In this manuscript, we will highlight the recent innovations in gene therapy for LSDs and discuss the clinical limitations that remain to be overcome, with the goal of fostering an understanding and further development of this important field. äRecent advances in gene therapy for lysosomal storage disorders (epmc).pdf DeepDyve Pubget Overpricing