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10.1101/cshperspect.a017376 http://scihub22266oqcxt.onion/10.1101/cshperspect.a017376 C4208711!4208711!25167981     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=25167981&cmd=llinks): Failed to open stream: HTTP request failed! HTTP/1.1 429 Too Many Requests in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 215 Deprecated: Implicit conversion from float 211.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 211.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534 Deprecated: Implicit conversion from float 211.6 to int loses precision in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 534       Cold+Spring+Harb+Perspect+Med 2014 ; 4 (11): ä Nephropedia Template TP {{tp|p=25167981|t=2014. Gene Therapy for PRPH2-Associated Ocular Disease: Challenges and Prospects |pdf=|usr=}}{{25167981}} gab.com Text Gene Therapy for PRPH2-Associated Ocular Disease: Challenges and Prospects JO: Cold Spring Harb Perspect Med http://www.kidney.de/pget.php?&tw=1&pmid=25167981 #FOAvip The peripherin-2 (PRPH2) gene encodes a photoreceptor-specific tetraspanin protein called peripherin-2/retinal degeneration slow (RDS), which is critical for the formation and maintenance of rod and cone outer segments. Over 90 different disease-causing mutations in PRPH2 have been identified, which cause a variety of forms of retinitis pigmentosa and macular degeneration. Given the disease burden associated with PRPH2 mutations, the gene has long been a focus for preclinical gene therapy studies. Adeno-associated viruses and compacted DNA nanoparticles carrying PRPH2 have been successfully used to mediate improvement in the rds?/? and rds+/? mouse models. However, complexities in the pathogenic mechanism for PRPH2-associated macular disease coupled with the need for a precise dose of peripherin-2 to combat a severe haploinsufficiency phenotype have delayed the development of clinically viable genetic treatments. Here we discuss the progress and prospects for PRPH2-associated gene therapy. Twit Text FOAVip Gene Therapy for PRPH2-Associated Ocular Disease: Challenges and Prospects ????Cold Spring Harb Perspect Med http://www.kidney.de/pget.php?&tw=1&pmid=25167981 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=25167981 #FOAvip English Wikipedia <ref>{{Cite pmid|25167981}}</ref> Gene Therapy for PRPH2-Associated Ocular Disease: Challenges and Prospects #MMPMID25167981 Conley SM; Naash MI Cold Spring Harb Perspect Med 2014[Nov]; 4 (11): ä PMID25167981show ga The peripherin-2 (PRPH2) gene encodes a photoreceptor-specific tetraspanin protein called peripherin-2/retinal degeneration slow (RDS), which is critical for the formation and maintenance of rod and cone outer segments. Over 90 different disease-causing mutations in PRPH2 have been identified, which cause a variety of forms of retinitis pigmentosa and macular degeneration. Given the disease burden associated with PRPH2 mutations, the gene has long been a focus for preclinical gene therapy studies. Adeno-associated viruses and compacted DNA nanoparticles carrying PRPH2 have been successfully used to mediate improvement in the rds?/? and rds+/? mouse models. However, complexities in the pathogenic mechanism for PRPH2-associated macular disease coupled with the need for a precise dose of peripherin-2 to combat a severe haploinsufficiency phenotype have delayed the development of clinically viable genetic treatments. Here we discuss the progress and prospects for PRPH2-associated gene therapy. äGene Therapy for PRPH2-Associated Ocular Disease: Challenges and Prosp(epmc).pdf DeepDyve Pubget Overpricing