Standardizing Genome Editing Workflows in the Lab

Lab Organization

Over the next decade, genome editing technology is expected to become a major factor in biomedical research and drug development. Image Credit: Flickr user Mehmet Pinarci

A March 2017 market analysis released by Grand View Research predicts that the genome editing market in the United States is expected to grow to $8.1 billion by 2025. According to the report, the most significant driver will be the increased use of the technology in the life sciences industry.1  As genome editing technology improves, scientists are finding more ways to apply the technology in the lab, from basic biomedical research to disease treatment development to biologics manufacturing. If your organization is looking to bring genome editing technology into the lab to support your research and development goals, it can be helpful to use modern software to standardize workflows in order to streamline the integration process.

Developing Innovative Treatment Methods

According to the Grand View Research market report, the primary driver of growth in the genome editing market over the next decade will be the development of therapeutics that can be used to treat disease. Here are some of the treatment strategies that scientists are exploring:

 

  • Ex-Vivo Genome Editing Therapy

 

So far ex-vivo treatment methods have the most demonstrated success, so they are likely to generate the largest share of the revenue over the next decade. Current ex-vivo genome editing therapy research focuses on the treatment of genetic blood and immune disorders. In these treatments, blood or immune cells are removed from the patient, genome editing technology is used to correct genetic abnormalities, and then the cells are returned to the body. Some of the diseases that may be treated using this method include leukemia, MRSA, muscular dystrophy, and AIDS.2

 

  • In-Vivo Genome Editing Therapy

 

Because many disease-related tissues are incompatible with cell transplantation, scientists are becoming increasingly focused on finding vectors to facilitate in-vivo genome editing. One possible vector is adeno-associated virus (AAV), which was recently used to demonstrate the effectiveness of nuclease-mediated gene editing in the liver in a mouse model of hemophilia B.3 However, research in this area is still in its infancy, so the field remains wide open for further studies on potential therapeutics.

 

  • Immune System Targeting

 

Rather than editing out genetic abnormalities in body cells (as in most ex-vivo and in-vivo genome editing treatment proposals), some scientists are looking to use genome editing to alter the way that the immune system responds to disease processes. Possible immune system targets include the C-C chemokine receptor type 5 and the programmed death 1 gene. Scientists are also looking to develop chimeric antigen receptor sin T cells that can support antitumor immunotherapy.4

 

  • Altering Pathogen Targets

 

Another area of research interest is to use genome editing to alter the molecular targets of pathogens, with the goal of reducing patient susceptibility to particular pathogens. This therapeutic concept could have applications in both disease treatments and vaccination options.

In addition to coming up with novel treatment methods, life science organizations that focus on therapeutics development can also explore how genome editing may be used to reduce side effects or improve the efficacy of existing therapeutics. Genome editing can also be used to improve the efficiency of biologics manufacturing.

Advancing Biomedical Research

Life science organizations focusing specifically on biomedical research can also benefit from genome editing technology. So far, the main use of genome editing in scientific research has been to create more accurate cell and animal models of disease. However, it can also be used to alter particular molecules in signaling cascades in order to better understand their functions in natural biological and disease processes, which can highlight potential drug targets. On a large scale, scientists are harnessing CRISPR gene editing technology to create pooled CRISPR guide RNA libraries (gRNA libraries), which can then be used for high-throughput screenings to identify possible pharmaceutical drug targets.5

Bringing Genome Editing Technology Into the Lab

With so many possible applications of genome editing technology, integrating it into a lab can lead to a decline in productivity. Researchers have to get used to the complex workflows associated with genome editing, and it is essential to standardize all processes in order to ensure that the results are repeatable. For research staff, it can be especially confusing if genome editing is being used for multiple purposes (for instance, the development of both ex-vivo and in-vivo treatment methods), since some of the associated workflows may differ.

One way to streamline the introduction of genome editing technology in the lab is to use modern workflow standardization software. Today’s technology makes it possible to lay out each step of a process using an authoring program. Researchers can access the protocol from mobile devices as they move around the lab, cutting down on accidental transcription errors and non-compliance issues. As a result, it won’t take long for labs to achieve a high level of efficiency, even given the complexities of today’s genome editing technologies.

BIOVIA Compose is a workflow authoring tool that can be used to define and standardize critical lab processes associated with genome editing. It can be paired with BIOVIA Capture, a mobile-compatible application that facilitates efficient process execution. Contact us today to learn more about these and our other software offerings!

  1. “Genome Editing Market Analysis by Technology,” March 29, 2017, https://finance.yahoo.com/news/genome-editing-market-analysis-technology-194200555.html
  2.  “What Is Genome Editing?” November 7, 2016, http://www.yourgenome.org/facts/what-is-genome-editing
  3. “Genome-editing Technologies for Gene and Cell Therapy,” March 2016, http://www.sciencedirect.com/science/article/pii/S1525001616309613
  4. “CRISPR-mediated Genome Editing and Human Diseases,” December 2016, http://www.sciencedirect.com/science/article/pii/S2352304216300356
  5. “CORALINA: a universal method for the generation of gRNA libraries for CRISPR-based screening,” November 14, 2016, https://bmcgenomics.biomedcentral.com/articles/10.1186/s12864-016-3268-z