Genetic variation among individual humans occurs on many different scales, ranging from
gross alterations in the human karyotype to single nucleotide changes. Here we explore …

The mouse (Mus musculus) is the premier animal model for understanding human disease
and development. Here we show that a comprehensive understanding of mouse biology is …

BACKGROUND Gene and engineered-cell therapies promise new treatment modalities for
incurable or difficult-to-treat diseases. First-generation gene and engineered-cell therapies …

Variation in genome structure is an important source of human genetic polymorphism: It affects
a large proportion of the genome and has a variety of phenotypic consequences relevant …

Engineering mammalian cell lines that stably express many transgenes requires the precise
insertion of large amounts of heterologous DNA into well-characterized genomic loci, but …

There is a complex relationship between the evolution of segmental duplications and
rearrangements associated with human disease. We performed a detailed analysis of one region …

Throughout biology, function is intimately linked with form. Across scales ranging from
subcellular to multiorganismal, the identity and organization of a biological structure’s subunits …

Synthetic mRNA is an attractive vehicle for gene therapies because of its transient nature
and improved safety profile over DNA. However, unlike DNA, broadly applicable methods to …

INTRODUCTION Synthetic biology applies engineering methodologies to biology to build
new functions into living cells. Through the assembly and characterization of engineered …

Self-replicating (replicon) RNA is a promising new platform for gene therapy, but applications
are still limited by short persistence of expression in most cell types and low levels of …