Gene therapy

Consistent cell count & analysis for best product control

Gene therapy is a new form of therapy and is still considered experimental for the most part, since clinical trials started as recently as 2017. There are two approaches to gene therapy: Specific genes are inserted into patient cells to: (1) overcome a genetic disorder that is causing disease, or (2) add new properties to cells by allowing the production of new molecules which can act as drugs against the disease. Gene therapy can be done on either somatic cells or germline cells, the former being the more common choice.

The recent advances in gene-editing tools such as CRISPR-Cas9, Lentiviral and Attenuated Adenoviral based tools have greatly increased the efficacy of editing the chromosome of cells in very specific ways, allowing for the development of new cell lines for various preclinical studies—targeting everything from cancers to genetic disorders. The specificity of these new tools also allows for their use in developing gene therapies to correct these genetic disorders at their source.

Mechanical arms that are about to edit a DNA molecule.

What’s the challenge?

Gene therapy can be performed in vivo, where genes of interest are introduced directly into patient cells in the body. Here, the primary hurdle is ensuring the gene of interest is introduced efficiently into the entire targeted cell population. Cell culture model systems that mimic tissue for in vivo gene therapies can be incredibly helpful and accelerate the process of getting your therapy into clinical trials, however it can be challenging to properly count your cells when they are grown using these 2D and 3D culture methods.

Gene therapy can also be performed in vitro, where genes of interest are introduced or manipulated in the lab. Once the cells are confirmed to be working as intended, they are introduced back into the patient, as is the case with cell-based immunotherapies using CAR-T/CAR-NK cells. For these in vitro methods, it is critical to know that your process has not affected the health of your cells and that the material introduced back into the patient is alive and active.

Solutions for gene therapy

When generating and validating a gene therapy, it is crucial to confirm numerous details of the manipulated cells, including their number and viability, and also proper expression and translation of the target gene.

The NucleoCounter® NC-202™ automated cell counter guarantees fast, easy and robust determination of cell numbers and viability with protocols that ensure reliable measurements of your cells, no matter the culture method.

With the NucleoCounter® NC-3000™, you can run up to eight analyses at a time, inspecting protein expression and cell phenotypes. In addition, our range of assays, including the Cell Cycle Assay and GFP Transfection Assay, give you an in-depth look at the health of your cells.