By Scott Magness, Ph.D., Altis Biosystems
Disease modeling provides the best results when actual human tissues are used. However, obtaining the full diversity of tissue types and capturing the variation of human cellular responses to pharmaceuticals in a dish is difficult. Accessing normal, healthy tissue to use as controls is one primary issue. Another, even more challenging issue is accessing tissues from many different people that have varied genetic backgrounds, which many times influence disease penetrance or drug efficacies. As a result, tissue access represents a bottleneck and a substantial challenge for disease modeling using tissue culture. Ultimately, the lack of this precious resource negatively impacts preclinical studies trying to evaluate drug efficacy
across the human population or disease cohort.
The Potential of Genetic Engineering
Genetic engineering provides a novel opportunity to model diseases. Gene-editing techniques, such as the CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9) technology and associated variants, can be applied to introduce disease-causing mutations into stem cells in a controlled manner. If tissues are available from individuals with appropriate genetic diversity, the consequences of specific mutations superimposed onto different genetic backgrounds can be observed, providing a better understanding of how drugs will perform across a population with a certain disease.
Read how gene editing enables Altis Biosystems to off er a diverse array of RepliGut® human gut models tailored for specific research programs.