Research

Research Interest

  • Optimize next generation AAV expression cassette
  • Development of viral strategies for targeting the central and peripheral nervous systems.
  • Applications and development of gene therapy drug delivery systems 
Tres marias

 

Main Research: Graphical Abstracts

We describe and validate three small pan-neuronal promoters isolated from the genome of the alphaherpesvirus, using neuron primary cells culture and mouse model. Promoters drive efficient and long-term transgene expression in the central nervous system after single-dose administration. For more details, check out our paper.

Paper1

We validated a short (404 bp), strong and persistent promoter obtained from the genome of pseudorabies virus (PRV) called alphaherpesvirus latency-associated promoter 2 (LAP2) in peripheral tissue. We demonstrate that different cell types can efficiently express a transgene when transduced by AAV8-LAP2 or AAV9-LAP2 following local or systemic administration. Here is our new article published on Journal Virological Methods.                                                                                               

We developed an assay for single-cell quantification of incoming AAV genome in neurons through a mathematical model using neuron primary cells culture and mouse model. Methodology and results can be found in our paper.

Paper2

We design a protocol for A) construction of triple-AAV plasmid, B) production of AAV vectors, C) triple-AAV transduction of primary superior cervical ganglia (SCG) neuronal cultures, mouse surgery and triple-AAV injection, image analysis and AAV genome quantification with single-cell resolution. Chek out our paper.

Current protocols
We examine the formulation buffers that viral core facilities typically use for cold AAV storage. Our findings confirm that AAV in the buffer that is composed of both Pluronic F68 and 5% glycerol was more stable after storage at 4°C and after several freeze/thaw cycles. For more details, check out our paper.
Angela paper

We Introduced MiniLAP2, a compact neural promoter enabling precise transgene expression in brain. Minimal off-target effects observed in peripheral tissues. Promising potential for safe and effective therapeutic delivery by AAV vector. Here is our new article published on Gene Therapy

Gene Therapy

We assessed the efficacy of LAP2, a compact promoter derived from pseudorabies virus, for driving transgene expression compared to the conventional EF1α promoter in mice after one year (400 days) post administration. Injecting LAP2 and EF1α packaged into AAV8 and AAV9 vectors, we observed robust transgene expression in liver, kidney, and skeletal muscle, suggesting LAP2's potential for gene therapy applications requiring efficient delivery of large or multiple therapeutic genes for long-term. Here is our latest article published in Frontier in Virology

Last paper