A5025571012- CRISPR and Genetic Engineering
- RNA and protein synthesis mechanisms
- Advanced biosensing and bioanalysis techniques
- RNA Interference and Gene Delivery
- RNA Research and Splicing
- RNA regulation and disease
- RNA modifications and cancer
- Cytomegalovirus and herpesvirus research
- Virus-based gene therapy research
- Mosquito-borne diseases and control
- Innovation and Socioeconomic Development
- Pluripotent Stem Cells Research
- MicroRNA in disease regulation
- Plant Virus Research Studies
- Viral Infections and Immunology Research
- Bacterial Genetics and Biotechnology
- Genetics, Aging, and Longevity in Model Organisms
- Bacteriophages and microbial interactions
- Insect symbiosis and bacterial influences
- Ubiquitin and proteasome pathways
- Chromosomal and Genetic Variations
- Animal Genetics and Reproduction
- Prion Diseases and Protein Misfolding
- DNA and Nucleic Acid Chemistry
- Biomedical Ethics and Regulation
University of Massachusetts Chan Medical School
2016-2025
Inspire
2023
Institute of Molecular Medicine
2022
Northwestern University
2006-2015
Howard Hughes Medical Institute
1992-2000
University of Chicago
1999-2000
Yale University
1992-1994
Hospital de Sant Pau
1992
Horizontal gene transfer (HGT) in bacteria and archaea occurs through phage transduction, transformation, or conjugation, the latter is particularly important for spread of antibiotic resistance. Clustered, regularly interspaced, short palindromic repeat (CRISPR) loci confer sequence-directed immunity against phages. A clinical isolate Staphylococcus epidermidis harbors a CRISPR spacer that matches nickase present nearly all staphylococcal conjugative plasmids. Here we show interference...
Genome engineering in human pluripotent stem cells (hPSCs) holds great promise for biomedical research and regenerative medicine. Recently, an RNA-guided, DNA-cleaving interference pathway from bacteria [the type II clustered, regularly interspaced, short palindromic repeats (CRISPR)-CRISPR-associated (Cas) pathway] has been adapted use eukaryotic cells, greatly facilitating genome editing. Only two CRISPR-Cas systems (from Streptococcus pyogenes thermophilus), each with their own distinct...
CRISPR/Cas9 derived from the bacterial adaptive immunity pathway is a powerful tool for genome editing, but safety profiles of in vivo delivered Cas9 (including host immune responses to protein) have not been comprehensively investigated model organisms. Nonalcoholic steatohepatitis (NASH) prevalent human liver disease characterized by excessive fat accumulation liver. In this study, we used adenovirus (Ad) vector deliver Streptococcus pyogenes–derived system (SpCas9) targeting Pten, gene...
Abstract Prime editors (PEs) mediate genome modification without utilizing double-stranded DNA breaks or exogenous donor as a template. PEs facilitate nucleotide substitutions local insertions deletions within the based on template sequence encoded prime editing guide RNA (pegRNA). However, efficacy of in adult mice has not been established. Here we report an NLS-optimized SpCas9-based editor that improves efficiency both fluorescent reporter cells and at endogenous loci cultured cell lines....
Five small nuclear RNAs (U1, U2, U4, U5, and U6) participate in precursor messenger RNA (pre-mRNA) splicing. To probe their interactions within the active center of mammalian spliceosome, substrates containing a single photoactivatable 4-thiouridine residue adjacent to either splice site were synthesized, crosslinks induced during course vitro An invariant loop sequence U5 contacts exon 1 before after first step splicing because crosslink between last appeared pre-mRNA then cutoff...
J R Wyatt, E Sontheimer, and A Steitz Department of Molecular Biophysics Biochemistry, Yale University School Medicine, Howard Hughes Medical Institute, Boyer Center for New Haven, Connecticut 06536-0812.
CRISPR is widely used to disrupt gene function by inducing small insertions and deletions. Here, we show that some single-guide RNAs (sgRNAs) can induce exon skipping or large genomic deletions delete exons. For example, CRISPR-mediated editing of β-catenin 3, which encodes an autoinhibitory domain, induces partial the in-frame nuclear accumulation β-catenin. A single sgRNA partially alter splicing unexpected larger remove Exon adds outcomes must be accounted for, perhaps taken advantage of,...