A5012951558- CRISPR and Genetic Engineering
- Virus-based gene therapy research
- Acute Myeloid Leukemia Research
- Cytomegalovirus and herpesvirus research
- Bacteriophages and microbial interactions
- Erythrocyte Function and Pathophysiology
- RNA and protein synthesis mechanisms
- Epigenetics and DNA Methylation
- Viral gastroenteritis research and epidemiology
- Chemistry and Stereochemistry Studies
- Cancer-related molecular mechanisms research
- Single-cell and spatial transcriptomics
- Molecular Biology Techniques and Applications
- Plant and Biological Electrophysiology Studies
- Biomedical Text Mining and Ontologies
- Bioinformatics and Genomic Networks
- RNA Interference and Gene Delivery
- CAR-T cell therapy research
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Cellular Mechanics and Interactions
- Animal Genetics and Reproduction
- Cancer Genomics and Diagnostics
- Genomics and Rare Diseases
- RNA regulation and disease
- Genomics and Phylogenetic Studies
National Institutes of Health
2017-2024
National Heart Lung and Blood Institute
2017-2024
Georgetown University
2013-2021
Georgetown University Medical Center
2013-2018
Center for Biologics Evaluation and Research
2017-2018
United States Food and Drug Administration
2017-2018
Duke University
2013
Jackson Laboratory
2013
University of Delaware
2013
Detection of distantly related viruses by high-throughput sequencing (HTS) is bioinformatically challenging because the lack a public database containing all viral sequences, without abundant nonviral which can extend runtime and obscure hits. Our reference (RVDB) includes viral, virus-related, virus-like nucleotide sequences (excluding bacterial viruses), regardless length, with overall reduced cellular sequences. Semantic selection criteria (SEM-I) were used to select from GenBank,...
The Protein Ontology (PRO; http://proconsortium.org) formally defines protein entities and explicitly represents their major forms interrelations. represented in PRO corresponding to single amino acid chains are categorized by level of specificity into family, gene, sequence modification metaclasses, there is a separate metaclass for complexes. All metaclasses also have organism-specific derivatives. complements established databases such as UniProtKB, interoperates with other biomedical...
Efficient and homogeneous in vitro generation of peripheral sensory neurons may provide a framework for novel drug screening platforms disease models touch pain. We discover that, by overexpressing NGN2 BRN3A, human pluripotent stem cells can be transcriptionally programmed to differentiate into surprisingly uniform culture cold- mechano-sensing neurons. Although such neuronal subtype is not found mice, we identify molecular evidence its existence ganglia. Combining BRN3A programming with...
Nonhuman primate (NHP) induced pluripotent stem cells (iPSCs) offer the opportunity to investigate safety, feasibility, and efficacy of proposed iPSC-derived cellular delivery in clinically relevant vivo models. However, there is need for stable, robust, safe labeling methods NHP iPSCs their differentiated lineages study survival, proliferation, tissue integration, biodistribution following transplantation. Here we utility adeno-associated virus integration site 1 (AAVS1) as a harbor...
Recent high-throughput sequencing (HTS) investigations have resulted in unexpected discoveries of known and novel viruses a variety sample types, including research materials, clinical biological products. Therefore, HTS can be powerful tool for supplementing current methods demonstrating the absence adventitious or unwanted products, particularly when using new cell line. However, is complex technology with different platforms, which needs standardization evaluation biologics. This...
The programmable nuclease technology CRISPR-Cas9 has revolutionized gene editing in the last decade. Due to risk of off-target editing, accurate and sensitive methods for characterization are crucial prior applying therapeutically. Here, we utilized a rhesus macaque model compare predictive values CIRCLE-seq, an vitro prediction method, with silico (ISP) based solely on genomic sequence comparisons. We use AmpliSeq HD error-corrected sequencing validate sites predicted by CIRCLE-seq ISP CD33...
ABSTRACT The programmable nuclease technology CRISPR/Cas9 has revolutionized gene editing in the last decade. Due to risk of off-target editing, accurate and sensitive methods for characterization are crucial prior applying therapeutically. Here, we utilized a rhesus macaque model ask whether CIRCLE-Seq (CS), an vitro prediction method, more accurately identifies off-targets compared silico (ISP) based solely on genomic sequence comparisons. We use AmpliSeq HD error-corrected sequencing...
In vitro generation of human peripheral sensory neurons may provide a framework for novel drug screening platforms and disease models touch pain. However, derivation functionally pure neuron population remains major unmet challenge. We discovered that, by expressing the transcription factors NGN2 BRN3A, pluripotent stem cells can be induced to differentiate into surprisingly homogenous culture cold- mechano-sensing neurons. Although such neuronal subtype has not been reported in mice, we...
Abstract For precise genome editing via CRISPR/homology-directed repair (HDR), effective and safe of long-term engrafting hematopoietic stem cells (LT-HSCs) requires both sufficient HDR efficiency protection LT-HSC function number. The impact on true LT-HSCs clonal dynamics in a relevant large animal model has not previously been studied. To track the HDR-edited cells, autologous rhesus macaque (RM) CD34 + were electroporated with gRNA/Cas9 ribonucleoprotein (RNP) cassette barcode library...
Individuals with age-related clonal hematopoiesis (CH) are at greater risk for hematologic malignancies and cardiovascular diseases, however, there no predictive preclinical animal models. We generated a rhesus macaque model via CRISPR/Cas9-mediated gene editing of hematopoietic stem progenitor cells (HSPCs), followed by autologous transplantation. Long-term follow-up revealed reproducible significant expansion multiple HSPC clones heterozygous TET2 loss-of-function mutations, compared to...