Gene Editing: Hype or Hope?
When our clients come to us to interrogate their genomic data, we never know what we might uncover. Pulling biological insights out of genetic data can range from identifying a single point mutation to entire extra copies of genes that may be implicated in the pathway or disease being investigated. However, once the mutation is identified, the challenging work of how to target that mutation begins.
What could change the game for developing genetics-based treatments would be the ability to edit the mutated DNA itself, rather than developing drugs targeting the downstream products of that mutation. However, gene editing is (understandably) difficult, even with the now ubiquitous CRISPR-Cas9 system. Considerations include ensuring that there is no off-target editing, delivering the CRISPR-Cas9 complex to the target cells, and whether genetic changes that can be inherited should be allowed.
CRISPR Engineered T Cells can Fight Cancer
In spite of the technical and ethical considerations of this technology, there is the potential for huge therapeutic benefit to patients. In an unusual move for the journal, Nature published a preprint last week of a paper outlining a new cancer therapy combining CRISPR gene editing and immunotherapy. Instead of directly editing mutated DNA, this approach leveraged gene editing to engineer a type of immune cell (T cells) to recognize and target specific cancer cells.
The therapeutic design is a huge bioinformatic feat. Samples were taken from each of the sixteen participants in the clinical trial and analyzed individually for the mutations specific to each person’s unique cancer. The researchers then ran the mutations through algorithms that predicted which mutations were the most likely to induce a strong T-cell-mediated immune response. Patients were then given immunosuppressants and infused with gene-edited versions of their own T cells that now expressed receptors for the mutated antigens unique to their cancer.
Five of the sixteen patients saw no progression in their cancer one month following treatment, and the authors hope that future iterations of the therapy will be more potent as the cells will have to spend less time being grown outside the body. This study is especially significant for solid tumors, which don’t express common surface proteins found on blood or lymph cancers that have been successfully targeted by CAR T therapy.
Future Directions for Precision Medicine
Precision medicine is an umbrella term for therapies that leverage a patient’s specific genetic information to be as effective as possible. While gene editing will likely have a place in this space as the technology continues to improve, there is a lot of research on precision approaches that may reach the clinic sooner. We covered two great examples in our most recent blogs, covering a direct nanopore sequencing-based diagnostic method for a unique class of genetic diseases as well as a predictive risk score based on the methylation pattern of the genome.
Outsourcing Bioinformatics Analysis: How We Can Help
The applications of precision medicine are innumerable, and our clients are at the forefront of tackling these research questions with sophisticated bioinformatics approaches. However, transforming raw sequence data of any kind into actionable biological insights is no small feat.
As experts across data types from cutting-edge sequencing platforms, we can help you tackle the challenging computational tasks of storing, analyzing and interpreting genomic data. Bridge Informatics’ bioinformaticians are trained bench biologists, so they understand the biological questions driving your computational analysis. Click here to schedule a free introductory call with a member of our team.
Jane Cook, Biochemist & Content Writer, Bridge Informatics
Jane Cook, leading Content Writer for Bridge Informatics, has written over 100 articles on the latest topics and trends for the bioinformatics community. Jane’s broad and deep interdisciplinary molecular biology experience spans developing biochemistry assays to genomics. Prior to joining Bridge, Jane held research assistant roles in biochemistry research labs across a variety of therapeutic areas. While obtaining her B.A. in Biochemistry from Trinity College in Dublin, Ireland, Jane also studied journalism at New York University’s Arthur L. Carter Journalism Institute. As a native Texan, she embraces any challenge that comes her way. Jane hails from Dallas but returns to Ireland any and every chance she gets. If you’re interested in reaching out, please email daniel.dacey@old.bridgeinformatics.com or dan.ryder@old.bridgeinformatics.com.
