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Genome Medicine

, 6:63

1. Genomics and epigenomics of disease


The development of the CRISPR-Cas system is revolutionizing genome editing in a variety of organisms. The system has now been used to manipulate the genome of Plasmodium falciparum, the most lethal malaria-causing species. The ability to generate gene deletions or nucleotide substitutions rapidly and economically promises to accelerate the analysis of novel drug targets and to help elucidate the function of specific genes or gene families, while complementing genome-wide association studies.

AbbreviationsCasCRISPR-associated proteins

CRISPRclustered regularly interspaced short palindromic repeats

DSBdouble-strand break

GFPgreen fluorescent protein

gRNAguide RNA

GWASgenome-wide association studies

NHEJnon-homologous end joining

PAMprotospacer adjacent motif

RBCred blood cell

RNAiRNA interference

snRNAsmall nuclear RNA

ZFNzinc-finger nuclease

Electronic supplementary materialThe online version of this article doi:10.1186-s13073-014-0063-9 contains supplementary material, which is available to authorized users.

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Autor: Marcus CS Lee - David A Fidock


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