BARRY JOHN BYRNE,CATHRYN MAH,CHRISTINA PACAK,DARIN J. FALK,DAVID D. FULLER,LARA ROBERT DERUISSEAU
申请号：
BR112014031788
公开号：
BR112014031788A2
申请日:
2013.06.19
申请国别(地区):
BR
年份:
2017
代理人:
摘要：
abstract the present invention provides compositions and methods of use pertaining to raav-mediated delivery of therapeutically effective molecules for treatment of diseases such as pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells. translation of the patent summary: "compositions and methods for treating diabetes". The present invention relates to compositions and methods of use pertaining to the application of therapeutically effective raav-mediated molecules for the treatment of diseases such as pomp disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.abstract the present invention provides compositions and methods of use pertaining to raav-mediated delivery of therapeutically effective molecules for treatment of diseases such as pompe disease. these compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells. tradução do resumo patente de invenção: "composições e métodos para o tratamento de diabetes". a presente invenção refere-se a composições e os métodos do uso que pertencem à aplicação de moléculas mediadas por raav terapeuticamente eficazes para o tratamento de doenças tais como a doença de pompe. estas composições em combinação com várias rotas e métodos de administração resultam na expressão visada de moléculas terapêuticas em órgãos, tecidos e células específicos.
