A5021517345- Virus-based gene therapy research
- Immune Cell Function and Interaction
- Genomics and Chromatin Dynamics
- SARS-CoV-2 and COVID-19 Research
- CAR-T cell therapy research
- Complement system in diseases
- SARS-CoV-2 detection and testing
- CRISPR and Genetic Engineering
- Viral-associated cancers and disorders
- Viral Infectious Diseases and Gene Expression in Insects
- RNA Interference and Gene Delivery
University of California, Los Angeles
2020-2024
We report the engineering of lipid nanoparticles (LNPs) to transport CRISPR/Cas9 payloads, including double-stranded DNA (dsDNA) donor templates, designed for homology directed repair (HDR)-mediated site-specific insertion cystic fibrosis transmembrane conductance regulator (CFTR) gene correct (CF) in diseased airway epithelium. screened various nanoparticle formulations, adjusting ratios Cas9-encoding mRNA, single guide RNAs (sgRNAs), and dsDNA templates optimize editing using human...
Abstract Genetically modified, induced pluripotent stem cells (iPSCs) offer a promising allogeneic source for the generation of functionally enhanced, chimeric antigen receptor (CAR) T cells. However, signaling CARs during early cell development and removal endogenous required to prevent alloreactivity pose significant challenges production mature conventional CAR from iPSCs. Here, we show that TCR-null, CD8αβ can be efficiently generated iPSCs by engineering stage-specific onset expression...
Hematopoietic stem cell gene therapy is a promising approach for treating disorders of the hematopoietic system. Identifying combinations
The spike (S) glycoprotein of SARS-Cov-2 facilitates viral entry into target cells via the cell surface receptor angiotensin-converting enzyme 2 (ACE2). Third generation HIV-1 lentiviral vectors can be pseudotyped to replace native CD4 tropic envelope protein virus and thereby either limit or expand population. We generated a modified S pseudotype which efficiently transduced ACE2-expressing with high specificity contain minimal off-target transduction ACE2 negative cells. By utilizing...