Gene Editing

Cell and gene therapies have seen record growth due to their potential to address the underlying causes of genetic and acquired diseases. Non-viral delivery methods are increasing in popularity due to their safety, cost-effectiveness, reduction in manufacturing time, and flexibility compared to viral vector delivery methods. Electroporation is a simple, broadly applicable method capable of delivering molecules to a wide range of cell types for either cell or gene therapy. This article outlines the top advantages of using electroporation in cell and gene therapy and why it might be a good approach for your own research group.

Incorporating fluorescence-based cell sorting into your lab grants you powerful access to new research areas and rarer cell types without sacrificing efficiency or results. New, powerful techniques like CRISPR and single-cell analysis are enhanced by the isolation of specific cells. But isolating cell populations of the right quality and quantity is both challenging and time consuming. We offer some tips to set yourself up for success and get the most out of your sorts.

Successful chimeric antigen receptor- (CAR-) T cell therapy requires a target antigen that is unique to cancer cells. But what happens when there are no unique antigens? Researchers at Columbia University Medical Center addressed this problem by replacing healthy non-target cells with genetically modified versions lacking the CAR-T cell target. Their results, published in PNAS, may provide a new avenue for treatment of some types of cancer.

A recent publication showed that expression of a protein involved in homology-directed repair (HDR) can improve efficiency of CRISPR-Cas9 gene editing. Learn how manipulation of DNA repair pathways and improvements to gene editing workflows, such as the addition of cell sorting, can facilitate applications of CRISPR-Cas9 technology.

The advent of CRISPR as a gene editing tool appears to have revolutionized drug discovery and development in a very short time. CRISPR-Cas9 technology is also being utilized to target HIV, to mutate or cut out the provirus. Read about what’s new in potential treatments for HIV and the challenges facing the industry.

Sangamo BioSciences, Inc., located in Richmond, California, is developing HIV therapeutics toward a functional cure using their proprietary zinc finger DNA-binding protein technology. Droplet digital PCR (ddPCR™) — or third generation PCR — offers a superior solution as it can detect target sequence within a single molecule, making it a powerful technology for low copy event detection.