Base editing – a groundbreaking molecular biology technique pioneered by Professor David Liu at the Broad Institute of Harvard and MIT – offers a precise means to modify an organism’s DNA sequence with unprecedented accuracy. This innovative technology holds immense potential for addressing monogenic disorders resulting from single nucleotide mutations, like sickle cell anemia, hemophilia, and cystic fibrosis. The creation of stable cell lines with specific genetic attributes is a crucial component of base editing. Professor Liu and his team relied on the SH800 in their seminal work to characterize and isolate cells with the desired attributes, and to validate stable gene expression. Base editing therapy has recently entered clinical trials for the first time in the US, marking a significant step forward in the progress of this revolutionary gene editing technology.
22q11.2 syndrome, also referred to as DiGeorge syndrome, is a genetic disorder resulting from the deletion of a small section of chromosome 22 in an individual’s DNA. This condition can lead to various symptoms, including heart defects, cleft palate, immune system issues, developmental delays, and psychiatric disorders. Despite DiGeorge syndrome’s significance, it has been relatively underexplored due to the lack of appropriate models that replicate the specific genome deletions involved.
Professor Arun Wiita and Dr. Neha Paranjape, in collaboration with Professor Mercedes Paredes, have used a CRISPRbased approach to address this challenge, successfully creating isogenic induced pluripotent stem (iPS) cell models. This approach allowed the University of California, San Francisco (UCSF) researchers to produce deletions that more accurately mimic the most commonly inherited variant of the disorder, taking advantage of the SH800 Cell Sorter to precisely isolate iPS cells with the necessary genome modifications. The cells were then differentiated into excitatory neurons, and in-depth analyses conducted using transcriptomics and cell surface proteomics. Significant proliferation and adhesion alterations were identified, which are associated with the specific deletion linked to the syndrome.
Paranjape, N., Lin, YH.T., Flores-Ramirez, Q. et al. A CRISPR-engineered isogenic model of the 22q11.2 A-B syndromic deletion. Sci Rep 13, 7689 (2023).https://doi.org/10.1038/s41598-023-34325-2
Explore our guide to streamlining and enhancing clone selection in CRISPR assays with the SH800 and MA900 cell sorters. The guide takes you through the key steps from initial sample preparation to precise system set-up. We also review basic instructions on how to maximize cell health throughout the sorting and post-sorting care stages. Our goal is to help you achieve a flawless and reliable clone selection and optimize your gene editing workflows.
Cell sorters have been used in combination with CRISPR to delve into gene regulation and intricate cellular mechanisms, and to enhance gene editing workflows. Cell sorters from Sony Biotechnology have been cited in over 1,000 scientific publications. Researchers frequently amalgamate sorting technologies with other cutting-edge tools such as single-cell sequencing and organoid systems to aid in-depth exploration of various biological and disease-related processes. These studies cover a broad range of crucial domains, including epigenetics, genome modification, cancer research, immunology, and stem cell biology – underscoring the indispensable contribution of CRISPR in advancing our knowledge and, potentially, opening doors to novel therapeutic possibilities.
Download our curated list of publications to immerse yourself in some of the most exciting CRISPR applications.
Did you know that the Sony Research Award Program supports cutting-edge academic research, fostering collaboration between faculty and Sony researchers? Grants can reach up to $150,000 USD per year, assisting pioneering research in various fields. We are proud to say that the 2023 Sony Research Award Program has supported numerous research institutions from all over the world.
October 2023 saw the launch of the Sony Innovation Fund: Africa, a $10 million fund to support early-stage startups in the gaming, music, movie, and content distribution sectors, which aims to boost the African entertainment industry’s growth and development.
