Applications of Gene Editing

By | December 15, 2023

Gene editing holds tremendous promise for improving human health through targeted modifications to our DNA. CRISPR-Cas9, the latest gene editing tool developed from bacterial immune systems, has enabled a new era of precision biomedical research and therapeutics. Here are some of the exciting ways scientists are applying this technology.

Treating and Curing Genetic Diseases
One major application is developing cures for inherited conditions caused by mutations in single genes. CRISPR shows promise for Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder affecting young boys (Hwang et al., 2020). DMD arises from mutations in the dystrophin gene, preventing production of this critical muscle protein. In animals, researchers have used CRISPR to successfully restore functional dystrophin, halting disease progression (Long et al., 2016). Clinical trials using CRISPR to treat DMD are launching soon. Other single-gene disorders CRISPR may cure include sickle cell anemia, cystic fibrosis, and some forms of childhood blindness (Porteus, 2020).

Fighting Cancer
Gene editing also targets complex diseases like cancer. Researchers at the Dana-Farber Cancer Institute have edited immune cells like T-cells using CRISPR/Cas9, knocking out PD-1 to boost anticancer function (Dana-Farber Cancer Institute, n.d.). Early clinical trials show promising results, with T-cells able to kill some hard-to-treat tumors in leukemia and lymphoma patients (Cui et al., 2021). Scientists also aim to disrupt oncogenes driving tumor growth or insert tumor-attacking immune cell receptors. Gene-edited cancer immunotherapy avoids harmful side effects of chemotherapy while offering personalized approaches.

gene therapy could help those with debilitating mental illnesses like schizophrenia, depression, or PTSD (Galbraith, 2020). Editing genes involved in serotonin and dopamine pathways may alleviate symptoms, complementing existing treatments.
Mitigating Genetic Disease Risk
CRISPR permits directly editing human embryos to prevent inherited conditions from being passed down. One day, parents carrying mutations for afflictions like Huntington’s disease or BRCA1 breast cancer variants could edit their embryos’ genomes before implantation (National Academies of Sciences, 2017). This remains controversial but could spare future generations from severe genetic illnesses. Another option lies in screening embryos for mutations and selectively implanting only healthy ones via in vitro fertilization, avoiding edits to the human germline (Nature Biotechnology, 2019).

Resources for Continued Learning:
The Alliance for Regenerative Medicine provides up-to-date gene and cell therapy clinical trial data: /
Addgene is a nonprofit DNA repository with numerous CRISPR-Cas9 plasmids for research use:
PubMed Central hosts full-text publications on gene editing applications:
The National Academies Press offers free reports evaluating emerging biotechnologies: