Making Gene Therapy More Cost-Efficient

There has been a lot of improvement in the area of gene editing and gene therapy in the medical industry, but recently scientists are trying to investigate how they can improve this field, and ultimately make it cheaper and more accessible. 

The first way therapy is being simplified is by using CRISPR-Cas9. CRISPR-Cas9 is a type of technology that lets medical researchers edit parts of the genome by removing, adding or changing sections of the DNA sequence. In a recent study in 2020, scientists edited the genetic makeup of a specialized subset of adult blood stem cells. The goal is to help people with blood diseases. 

Studies from the past have shown symptoms are reversed by reactivating a version of hemoglobin that works during fetal development, but is then turned off by our first birthdays. Therefore, they tried to make the fetal hemoglobin again. Researchers used the CRISPR-Cas9 gene editing to remove a piece of genetic code that normally turns off fetal hemoglobin proteins. They “snipped this control DNA with CRISPR, enabling red blood cells to continuously produce elevated levels of fetal hemoglobin” (source 1).The cells were then edited easily and efficiently, with different components of hemoglobin in the blood. This shows high potential because the “cells engraft efficiently at high levels, and this can help develop a more effective therapy for patients with blood diseases” (source 1). The scientists’ study showed that there were no harmful or off-target mutations in the cells. Carrying out genetic fixes on the smaller pool of cells required for therapeutic benefit will lessen safety concerns and reduce the risk of off-target effects overall. Since CRISPR is still a relatively new technology, it is important to ensure that it is safe, and that is being done through this study. 

Scientists also specifically experimented on adult blood stem cells to help reduce the cost of gene therapy. The self-renewing properties of these stem cells make them a powerful potential candidate to deliver gene therapy because they can provide long-term production of genetically modified blood cells and thus could cure a disease for an entire lifetime. Since they represent a mere 5% of all blood stem cells, targeting them with gene-editing machinery would require fewer supplies and potentially be less costly.

Another way cost is being reduced in the gene therapy field is through single-cell technologies, in which scientists learn about the molecular and more detailed characteristics of individual cells in a blood sample. These technologies give more detailed data to researchers, ultimately providing them with more knowledge. This can play a significant role in how efficient treatment is. 

Another initiative being taken is adding three-dimensional information to scientists’ single-cell analyses. These datasets are called “multi-modal datasets. This will help identify the cells that need to be modified more efficiently, and can speed up the process as well. In conclusion, scientists are using CRISPR, single cell technologies, and three-dimensional models to increase efficiency and lower cost of gene therapy. 

Sources:

• https://www.sciencedaily.com/releases/2019/07/190731145815.htm

• https://www.fredhutch.org/en/news/center-news/2020/02/future-medicine-aaas-panel.html