Genome engineering technologies provide an entry point into understanding and controlling
the function of genetic elements in health and disease. The discovery and development of …

While the catalog of mammalian transcripts and their expression levels in different cell types
and disease states is rapidly expanding, our understanding of transcript function lags …

Neurodegenerative, neurodevelopmental and neuropsychiatric disorders are among the
greatest public health challenges, as many lack disease-modifying treatments. A major …

An ideal in vivo gene therapy platform provides safe, reprogrammable, and precise
strategies which modulate cell and tissue gene regulatory networks with a high temporal …

Differences in genomes underlie most organismal diversity, and aberrations in genomes
underlie many disease states. With the growing knowledge of the genetic and pathogenic …

Large portions of the human genome harbor functional noncoding elements, which can
regulate a variety of biological processes and have important implications for disease risk …

Background Technologies for making and manipulating DNA have enabled advances in
biology ever since the discovery of the DNA double helix. But introducing site-specific …

Recent advances in genome engineering technologies based on the CRISPR-associated
RNA-guided endonuclease Cas9 are enabling the systematic interrogation of mammalian …

The CRISPR-Cas system is commonly known for its ability to cleave DNA in a
programmable manner, which has democratized gene editing and facilitated recent …

The discovery and adaption of bacterial clustered regularly interspaced short palindromic
repeats (CRISPR)–CRISPR-associated (Cas) systems has revolutionized the way …