The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …

Next-generation DNA sequencing technologies have led to a massive accumulation of
genomic and transcriptomic data from patients and healthy individuals. The major challenge …

Clustered regularly interspaced short palindromic repeat interference (CRISPRi), based on
the fusion of inactive Cas9 (dCas9) to the Krüppel-associated box (KRAB) repressor, is a …

Gene regulation plays a critical role in the cellular processes that underlie human health and
disease. The regulatory relationship between transcription factors (TFs), key regulators of …

The genetic interrogation and reprogramming of cells requires methods for robust and
precise targeting of genes for expression or repression. The CRISPR-associated …

The most widely used approach for defining gene function is to reduce or completely disrupt
its normal expression. For over a decade, RNAi has ruled the lab, offering a magic bullet to …

Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …

Targeted nucleases have provided researchers with the ability to manipulate virtually any
genomic sequence, enabling the facile creation of isogenic cell lines and animal models for …

Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by
a CAG trinucleotide expansion in the huntingtin gene (HTT), which codes for the pathologic …

Eukaryotic cells execute complex transcriptional programs in which specific loci throughout
the genome are regulated in distinct ways by targeted regulatory assemblies. We have …