For the roughly 30,000 Americans living with hemophilia and thousands more with rare enzyme deficiencies, treatment often means a lifetime of intravenous infusions, sometimes multiple times a week. A ...

Genome editing has become a widely adopted technology to modify DNA in cells, allowing scientists to study diseases in the lab and develop therapies that repair disease-causing mutations. However, ...

Mission Bio, the single-cell multi-omics leader, today released data showing that its Targeted Single-Cell DNA+RNA Assay simultaneously resolves CRISPR edit co-occurrence, allelic zygosity, and ...

PASSIGE installs edits in only a modest fraction of cells, which is enough to treat some but probably not most genetic diseases that result from the loss of a functioning gene. So Liu's team, in the ...