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Long-term Physiologically Regulated Expression of the Low-density Lipoprotein Receptor In Vivo Using Genomic DNA Mini-gene Constructs

Familial hypercholesterolemia (FH) is a condition caused by mutations in the low-density lipoprotein receptor (LDLR) gene. Expression of LDLR is highly regulated and excess receptor expression is cytotoxic. To incorporate essential gene regulation into a gene therapy vector for FH, we generated vect...

詳細記述

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書誌詳細
主要な著者: Hibbitt, Olivia C, McNeil, Eileen, Lufino, Michele MP, Seymour, Len, Channon, Keith, Wade-Martins, Richard
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group 2009
主題:
オンライン・アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839284/
https://ncbi.nlm.nih.gov/pubmed/19861949
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.249
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