Abstract mRNA-based drugs have tremendous potential as clinical treatments, however, a
major challenge in realizing this drug class will promise to develop methods for safely …

Viral delivery of DNA for the targeted reprogramming of human T cells can lead to random
genomic integration, and electroporation is inefficient and can be toxic. Here we show that …

Comparative genomics has enabled the discovery of tiny clustered regularly interspaced
short palindromic repeat (CRISPR) bacterial immune system effectors with enormous …

The editing efficiencies of prime editing (PE) using ribonucleoprotein (RNP) and RNA
delivery are not optimal due to the challenges in solid-phase synthesis of long PE guide …

Duplication is a foundation of molecular evolution and a driver of genomic and complex
diseases. Here, we develop a genome editing tool named Amplification Editing (AE) that …

Delivering ribonucleoproteins (RNPs) for in vivo genome editing is safer than using viruses
encoding for Cas9 and its respective guide RNA. However, transient RNP activity does not …

Reactivating silenced γ-globin expression through the disruption of repressive regulatory
domains offers a therapeutic strategy for treating β-hemoglobinopathies. Here, we used …

Despite the great potential of CRISPR-based detection, it has not been competitive with
other market diagnostics for on-site and in-home testing. Here we dissect the rate-limiting …

Combination therapy with PD-1 blockade and IL-2 substantially improves anti-tumor efficacy
comparing to monotherapy. The underlying mechanisms responsible for the synergistic …

Acute myeloid leukemia (AML) is a malignant cancer characterized by abnormal
differentiation of hematopoietic stem and progenitor cells (HSPCs). While chimeric antigen …