Ontology highlight
ABSTRACT:
SUBMITTER: Gulei D
PROVIDER: S-EPMC6639591 | biostudies-literature | 2019 Sep
REPOSITORIES: biostudies-literature
Gulei Diana D Berindan-Neagoe Ioana I
Molecular therapy. Nucleic acids 20190703
CRISPR/Cas9 has proved its efficiency in vitro, where we now know that this tool can efficiently target specific parts of the genome. These modifications can be used to generate advanced models of human diseases, address specific functions of genes, and develop new therapeutic strategies. Even if these advancements are promising, there are still two great issues associated with CRISPR/Cas9: how we can specifically and safely deliver the editing tool in vivo and how we can address the impossibili ...[more]