Ontology highlight
ABSTRACT:
SUBMITTER: Zabaleta N
PROVIDER: S-EPMC6303323 | biostudies-literature | 2018 Dec
REPOSITORIES: biostudies-literature
Zabaleta Nerea N Barberia Miren M Martin-Higueras Cristina C Zapata-Linares Natalia N Betancor Isabel I Rodriguez Saray S Martinez-Turrillas Rebeca R Torella Laura L Vales Africa A Olagüe Cristina C Vilas-Zornoza Amaia A Castro-Labrador Laura L Lara-Astiaso David D Prosper Felipe F Salido Eduardo E Gonzalez-Aseguinolaza Gloria G Rodriguez-Madoz Juan R JR
Nature communications 20181221 1
CRISPR/Cas9 technology offers novel approaches for the development of new therapies for many unmet clinical needs, including a significant number of inherited monogenic diseases. However, in vivo correction of disease-causing genes is still inefficient, especially for those diseases without selective advantage for corrected cells. We reasoned that substrate reduction therapies (SRT) targeting non-essential enzymes could provide an attractive alternative. Here we evaluate the therapeutic efficacy ...[more]