{"id":21507,"date":"2016-02-02T00:48:03","date_gmt":"2016-02-02T08:48:03","guid":{"rendered":"http:\/\/lifeboat.com\/blog\/2016\/02\/penn-study-has-implications-for-developing-safe-therapies-for-an-array-of-rare-diseases-via-new-gene-cut-and-paste-methods"},"modified":"2017-06-04T20:13:18","modified_gmt":"2017-06-05T03:13:18","slug":"penn-study-has-implications-for-developing-safe-therapies-for-an-array-of-rare-diseases-via-new-gene-cut-and-paste-methods","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2016\/02\/penn-study-has-implications-for-developing-safe-therapies-for-an-array-of-rare-diseases-via-new-gene-cut-and-paste-methods","title":{"rendered":"Penn study has implications for developing safe therapies for an array of rare diseases via new gene cut-and-paste methods"},"content":{"rendered":"<p><a class=\"blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/penn-study-has-implications-for-developing-safe-therapies-for-an-array-of-rare-diseases-via-new-gene-cut-and-paste-methods.jpg\"><\/a><\/p>\n<p>NGS \u2014 news flash; gene editing corrects genetically linked liver disease.<\/p>\n<hr>\n<p>For the first time, researchers have treated an animal model of a genetic disorder using a viral vector to deliver genome-editing components in which the disease- causing mutation has been corrected. Delivery of the vector to newborn mice improved their survival while treatment of adult animals, unexpectedly, made them worse, according to a new study by investigators from the Perelman School of Medicine at the University of Pennsylvania The team published their findings in <em>Nature Biotechnology<\/em>.<\/p>\n<p>\u201cCorrecting a disease-causing mutation following birth in this animal model brings us one step closer to realizing the potential of personalized medicine,\u201d said senior author James Wilson, MD, PhD, a professor of Medicine and director of the Orphan Disease Center at Penn. \u201cNevertheless, my 35-year career in gene therapy has taught me how difficult translating mouse studies to successful human treatments can be. From this study, we are now adjusting the gene-editing system in the next phases of our investigation to address the unforeseen complications seen in adult animals.\u201d Wilson is also director of the Penn Gene Therapy Program.<\/p>\n<p>The Wilson lab focused on liver as a target for gene editing since they had solved the problem of gene delivery in this organ in previous work using traditional gene therapy using vectors based on adeno-associated virus (AAV). However, gene replacement therapy with AAV is not ideal for treating genetic diseases of the liver that manifest as newborns since the non-integrating genome is lost as developing liver cells proliferate.<\/p>\n<p><a href=\"http:\/\/www.sciencedaily.com\/releases\/2016\/02\/160201122852.htm\" target=\"_blank\">Read more<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>NGS \u2014 news flash; gene editing corrects genetically linked liver disease. For the first time, researchers have treated an animal model of a genetic disorder using a viral vector to deliver genome-editing components in which the disease- causing mutation has been corrected. Delivery of the vector to newborn mice improved their survival while treatment of [\u2026]<\/p>\n","protected":false},"author":395,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11,412],"tags":[],"class_list":["post-21507","post","type-post","status-publish","format-standard","hentry","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/21507","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/395"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=21507"}],"version-history":[{"count":3,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/21507\/revisions"}],"predecessor-version":[{"id":68633,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/21507\/revisions\/68633"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=21507"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=21507"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=21507"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}