Scalable sequence–function studies have enabled the systematic analysis and cataloging of
hundreds of thousands of coding and noncoding genetic variants in the human genome …

Insulin-producing β cells created from human pluripotent stem cells have potential as a
therapy for insulin-dependent diabetes, but human pluripotent stem cell-derived islets (SC …

DNA double-strand breaks (DSBs) are among the most serious forms of DNA damage. In
humans, DSBs are repaired mainly by non-homologous end joining (NHEJ) and …

Resolving fundamental molecular and functional processes underlying human synaptic
development is crucial for understanding normal brain function as well as dysfunction in …

CRISPR-mediated transcriptional activation (CRISPRa) is a powerful technology for
inducing gene expression from endogenous loci with exciting applications in high …

Background The proximal tubule plays a major role in electrolyte homeostasis. Previous
studies have shown that HNF4A regulates reabsorption-related genes and promotes …

Human pluripotent stem cells (hPSCs) have proven to be valuable tools for both drug
discovery and the development of cell-based therapies. However, the long non-coding RNA …

The most widely used genome editing toolkit is CRISPR (clustered regularly interspaced
short palindromic repeats). It provides the possibility of replacing and modifying DNA and …

With the recent advancements in genome editing, next-generation sequencing (NGS), and
scalable cloning techniques, scientists can now conduct genetic screens at unprecedented …

Down syndrome (DS), driven by an extra copy of chromosome 21 (HSA21), and fragile X
syndrome (FXS), driven by loss of the RNA-binding protein FMRP, are two common genetic …