Human pluripotent stem cells (hPSCs) have potential applications in biological studies and regenerative medicine. However, precise genome editing in hPSCs remains time-consuming and labor-intensive. Here we demonstrate that the recently identified CRISPR-Cpf1 can be used to efficiently generate knockout and knockin hPSC lines. The unique properties of CRISPR-Cpf1, including shorter crRNA length and low off-target activity, are very attractive for many applications. In particular, we develop an unbiased drug-selection-based platform feasible for high-throughput screening in hPSCs and this screening system enables us to identify small molecules VE-822 and AZD-7762 that can promote CRISPR-Cpf1-mediated precise genome editing. Significantly, the combination of CRISPR-Cpf1 and small molecules provides a simple and efficient strategy for precise genome engineering.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5880812 | PMC |
| http://dx.doi.org/10.1038/s41467-018-03760-5 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Cell type-specific upregulation of NKG2D ligand MICA in response to APTO253.
Ann Transl Med
December 2024
Institute for Tumor Immunology, Center for Tumor Biology and Immunology, Philipps-University Marburg, Marburg, Germany.
One of the most important targets for natural killer (NK) cell-mediated therapy is the induction of natural killer group 2D ligand (NKG2D-L) expression. APTO253 is a small molecule that selectively kills acute myeloid leukemia (AML) cells, and it has been reported that APTO253 can induce Krüppel-like factor 4 (KLF4) expression and downregulate c-MYC expression. Recently, we discovered a novel role of APTO253 in modulating the NK cell response by inducing surface expression of NKG2D-Ls, especially MHC class I polypeptide-related sequence A (MICA), in AML cells.
View Article and Find Full Text PDFExpanding therapeutic options for Pompe disease: a new small molecule inhibitor of glycogen synthase 1 (GYS1) shows preclinical promise in Pompe disease.
Ann Transl Med
December 2024
Division of Medical Genetics, Duke University Medical Center, Durham, NC, USA.
Protein nanoparticles as potent delivery vehicles for polycytosine RNA-binding protein one.
World J Diabetes
January 2025
State Key Laboratory of Bioactive Molecules and Druggability Assessment, Guangdong Basic Research Center of Excellence for Natural Bioactive Molecules and Discovery of Innovative Drugs, College of Pharmacy, Jinan University, Guangzhou 511436, Guangdong Province, China.
Ma recently reported in the that ferroptosis occurs in osteoblasts under high glucose conditions, reflecting diabetes pathology. This condition could be protected by the upregulation of the gene encoding polycytosine RNA-binding protein 1 (PCBP1). Additionally, Ma used a lentivirus infection system to express PCBP1.
View Article and Find Full Text PDFIdentification of potent schistosomicidal compounds predicted as type II-kinase inhibitors against c-Jun N-terminal kinase SMJNK.
Front Parasitol
April 2024
Institut für Parasitologie, Biomedizinisches Forschungszentrum Seltersberg (BFS), Justus Liebig Universitaet Giessen, Giessen, Germany.
Introduction: Schistosomiasis has for many years relied on a single drug, praziquantel (PZQ) for treatment of the disease. Immense efforts have been invested in the discovery of protein kinase (PK) inhibitors; however, given that the majority of PKs are still not targeted by an inhibitor with a useful level of selectivity, there is a compelling need to expand the chemical space available for synthesizing new, potent, and selective PK inhibitors. Small-molecule inhibitors targeting the ATP pocket of the catalytic domain of PKs have the potential to become drugs devoid of (major) side effects, particularly if they bind selectively.
View Article and Find Full Text PDFInterleukin-17A facilitates tumor progression upregulating programmed death ligand-1 expression in hepatocellular carcinoma.
World J Gastrointest Oncol
January 2025
The First School of Clinical Medicine, Lanzhou University, Lanzhou 730000, Gansu Province, China.
Background: Hepatocellular carcinoma (HCC) is an inflammation-associated tumor with a dismal prognosis. Immunotherapy has become an important treatment strategy for HCC, as immunity is closely related to inflammation in the tumor microenvironment. Inflammation regulates the expression of programmed death ligand-1 (PD-L1) in the immunosuppressive tumor microenvironment and affects immunotherapy efficacy.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!