A5076608046- CRISPR and Genetic Engineering
- RNA Interference and Gene Delivery
- Advanced biosensing and bioanalysis techniques
- Cytomegalovirus and herpesvirus research
- RNA regulation and disease
- Virus-based gene therapy research
- RNA and protein synthesis mechanisms
- Genetics, Aging, and Longevity in Model Organisms
- Semiconductor materials and interfaces
- HIV Research and Treatment
- Cancer Mechanisms and Therapy
- Carbon Nanotubes in Composites
- Superconductivity in MgB2 and Alloys
- Chemical Synthesis and Characterization
- RNA modifications and cancer
- Ion-surface interactions and analysis
- Metal and Thin Film Mechanics
- Innovation and Socioeconomic Development
- RNA Research and Splicing
- Radioactive element chemistry and processing
- Protein Degradation and Inhibitors
- Pluripotent Stem Cells Research
- Covalent Organic Framework Applications
- Rare-earth and actinide compounds
The University of Texas Southwestern Medical Center
2025
University of Massachusetts Chan Medical School
2018-2024
National Institute for Radiological Protection
2023
Saint Louis University
2013
University of Electronic Science and Technology of China
2007
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but severely constrained by cargo limits. Simultaneous of multiple can limit dose and efficacy increase safety risks. Here, we describe single-vector, ~4.8-kb AAV that express Nme2Cas9 either two sgRNAs segmental deletions, or a single sgRNA with homology-directed repair (HDR) template. We also use anti-CRISPR proteins to enable production self-inactivate via cleavage. further introduce...
Nuclease-directed genome editing is a powerful tool for investigating physiology and has great promise as therapeutic approach to correct mutations that cause disease. In its most precise form, can use cellular homology-directed repair (HDR) pathways insert information from an exogenously supplied DNA-repair template (donor) directly into targeted genomic location. Unfortunately, particularly long insertions, toxicity delivery considerations associated with DNA limit HDR efficacy. Here, we...
Prime editing has gained significant attention as a next-generation gene technology, owing to its unique advantages. However, realizing potential in vivo requires effective delivery strategies. While adeno-associated virus (AAV) been employed for of prime editors research settings, it presents inherent limitations related vector size, ongoing expression, and inability re-dose patients. Conversely, lipid nanoparticles (LNPs) do not face these are emerging leading non-viral approach the...
The standard sodium concentration for RNA optical melting experiments is 1.021 M. Algorithms that predict Tm, ΔG°37, and secondary structure from sequence generally rely on parameters derived performed in M sodium. Physiological monovalent cation concentrations are much lower than In fact, many molecular biology techniques require buffers containing other Predictions based the Na+ may not be accurate when Here, we report thermodynamic data a set of 18 duplexes, each melted over wide range...
Abstract Prime editing efficiency is modest in cells that are quiescent or slowly proliferating where intracellular dNTP levels tightly regulated. MMLV-reverse transcriptase - the prime editor polymerase subunit requires high dNTPs for efficient polymerization. We report primary and vivo increased by mutations enhance enzymatic properties of can be further complemented targeting SAMHD1 degradation.
Abstract Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add challenges delivery. Chemical modification, which has been key to the success of oligonucleotide therapeutics, can enhance stability, distribution, cellular uptake, and safety nucleic acids. Previously, we engineered heavily fully modified SpyCas9 crRNA tracrRNA, showed enhanced stability retained activity when delivered cultured cells in form ribonucleoprotein complex. In this study, report that a short,...
Lanthanum hexaboride(LaB6) films have been deposited on silicon tip field emitters by electron-beam evaporation. The emission characteristics are studied in a diode test cell vacuum system. experimental results show that the stability of LaB6-coated Si-tip FEA can be improved and current is significantly enhanced to 75 µA, contrast pure 500 nA Mo-coated 300 with 1500 V applied anode. Furthermore, even at low (>1.5 × 10−5 Torr) it still exhibits good properties strong ability withstand ion...
Abstract Nuclease-directed genome editing is a powerful tool for investigating physiology and has great promise as therapeutic approach to correct mutations that cause disease. In its most precise form, can use cellular homology-directed repair (HDR) pathways insert information from an exogenously supplied DNA template (donor) directly into targeted genomic location. Unfortunately, particularly long insertions, toxicity delivery considerations associated with limit HDR efficacy. Here, we...
CRISPR-Cas technology has revolutionized genome editing. Its broad and fast-growing application in biomedical research therapeutics led to increased demand for guide RNAs. The synthesis of chemically modified single-guide RNAs (sgRNAs) containing >100 nucleotides remains a bottleneck. Here we report the development tetrazine ligation method preparation sgRNAs. A moiety on 3′-end crRNA norbornene 5′-end tracrRNA enable successful between form sgRNA under mild conditions. Tetrazine-ligated...
CRISPR-based genome-editing technologies, including nuclease editing, base and prime have recently revolutionized the development of therapeutics targeting disease-causing mutations. To advance assessment genome editing tools, a robust mouse model is valuable, particularly for evaluating
Abstract CRISPR-Cas genome editing tools enable precise, RNA-guided modification of genomes within living cells. The most clinically advanced editors are Cas9 nucleases, but many nuclease technologies provide only limited control over outcomes. Adenine base (ABEs) and cytosine (CBEs) precise efficient nucleotide conversions A:T-to-G:C C:G-to-T:A pairs, respectively. Therapeutic use (BEs) provides an avenue to correct approximately 30% human pathogenic variants. Nonetheless, factors such as...
Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add challenges delivery. Chemical modification, which has been key to the success of oligonucleotide therapeutics, can enhance stability, distribution, cellular uptake, and safety nucleic acids. Previously, we engineered heavily fully modified SpyCas9 crRNA tracrRNA, showed enhanced stability retained activity when delivered cultured cells in form ribonucleoprotein complex. In this study, report that a short, stabilized...