Methods for the production of functional protein from DNA having a nonsense mutation and the treatment of disorders associated therewith
摘要
<p>The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.</p>
申请公布号
AU2007235524(B2)
申请公布日期
2013.09.12
申请号
AU20070235524
申请日期
2007.03.29
申请人
PTC THERAPEUTICS, INC.
发明人
REN, HONGYU;HIRAWAT, SAMIT;WELCH, ELLEN M.;WILDE, RICHARD G.;ALMSTEAD, NEIL G.;KARP, GARY M.;MOON, YOUNG-CHOON;TAKASUGI, JEMES J.;MILLER, LANGDON;CHEN, GUANGMING;HWANG, SEONGWOO