A5037518548- CRISPR and Genetic Engineering
- Virus-based gene therapy research
- Microbial Inactivation Methods
- Microfluidic and Bio-sensing Technologies
- Mosquito-borne diseases and control
- RNA Interference and Gene Delivery
Rice University
2022-2025
Homology-directed repair (HDR)–based genome editing is an approach that could permanently correct a broad range of genetic diseases. However, its utility limited by inefficient and imprecise DNA mechanisms in terminally differentiated tissues. Here, we tested Repair Drive, platform technology for selectively expanding HDR-corrected hepatocytes adult mice vivo. Drive involves transient conditioning the liver knocking down essential gene, fumarylacetoacetate hydrolase ( Fah ), delivering...
Most genome editing analyses to date are based on quantifying small insertions and deletions. Here, we show that CRISPR-Cas9 can induce large gene modifications, such as deletions, insertions, complex local rearrangements in different primary cells cell lines. We analyzed deletion events hematopoietic stem progenitor (HSPCs) using methods, including clonal genotyping, droplet digital polymerase chain reaction, single-molecule real-time sequencing with unique molecular identifier,...
Manipulation of cells for applications such as biomanufacturing and cell-based therapeutics involves introducing biomolecular cargoes into cells. However, successful delivery is a function multiple experimental factors requiring several rounds optimization. Here, we present high-throughput multiwell-format localized electroporation device (LEPD) assisted by deep learning image analysis that enables quick optimization efficient delivery. We showcase the versatility LEPD platform successfully...