Induced pluripotent stem cell (iPSC)-derived organoids provide models to study human organ development. Single-cell transcriptomics enable highly resolved descriptions of cell …
Rationale: Loss-of-function studies in cardiac myocytes (CMs) are currently limited by the need for appropriate conditional knockout alleles. The factors that regulate CM maturation …
To the Editor—The field of genome editing is advancing rapidly1, most recently exemplified by the advent of base editors that enable changing single nucleotides in a predictable …
S Riesenberg, P Kanis, D Macak, D Wollny… - Nature …, 2023 - nature.com
Abstract Homology-directed repair (HDR), a method for repair of DNA double-stranded breaks can be leveraged for the precise introduction of mutations supplied by synthetic DNA …
CRISPOR. org is a web tool for genome editing experiments with the CRISPR–Cas9 system. It finds guide RNAs in an input sequence and ranks them according to different scores that …
In the human malaria vector Anopheles gambiae, the gene doublesex (Agdsx) encodes two alternatively spliced transcripts, dsx-female (AgdsxF) and dsx-male (AgdsxM), that control …
The diverse malignant, stromal, and immune cells in tumors affect growth, metastasis, and response to therapy. We profiled transcriptomes of∼ 6,000 single cells from 18 head and …
CE Nelson, Y Wu, MP Gemberling, ML Oliver… - Nature medicine, 2019 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in …