There is a new gene editing therapy approach under use by a team of researchers at Yale University that can correct mutations that cause a form of anemia called thalassemia. If their findings are proven to be effective, scientists may be able to harness similar gene correction therapy methods to develop treatments for people who suffer from other blood disorders.
Gene Editing and Inherited Disease
Occasionally, people are born with abnormalities in their genetics that can lead to certain types of anemia. Some of the most common are:
- Sickle cell anemia
- Thalassemia
- Fanconi anemia
Some types of anemia develop because of poor eating habits or other lifestyle factors, but inherited anemia is strikingly common. For those who have such ailments, lifelong management is necessary for a prolonged life.
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| Thalassemia diagram. Source: nih.gov |
In answer to the misfortune of hereditary anemia, researchers have been developing the technique of gene editing therapy for mutated cells in laboratories that grow cell clusters, but their research has not moved on to living animals until recently. A team of researchers led by Peter M. Glazer, M.D. developed an approach to gene editing therapy in mice with thalassemia using synthetic pieces of DNA and nanoparticles with an intravenous (IV) injection.
The goal is to trigger the natural repair process in cells by combining protein derived from bone marrow with DNA-like synthetic molecules called PNA. The nanoparticles, a development of professor of biomedical engineering Mark Saltzman and his lab, transport PNAs to the mutation. The team combined the two processes in an IV injection to improve targeting.
Results in Tests with Mice
Using their gene correction therapy technique, the team managed to correct mutations so well that the mice stopped displaying symptoms of thalassemia. They returned to test the mice after 140 days and found that their hemoglobin levels had returned to normal.
Compared to other techniques, such as the CRISPR technique that takes a more aggressive approach to genome alteration, unintended effects appeared minimally. The team declared that they had achieved gene editing therapy to an effective enough degree to cure anemia in mice with thalassemia.
Gene Editing Therapy and Hereditary Ailments in the Future
Similar tests will move onto clinical studies so that researchers can study the possible effects of gene editing on humans who have thalassemia. If successful, it may open doors to the study of gene correction therapy’s efficacy in related hereditary blood disorders such as sickle cell anemia.
However, mutation correction is a resource-heavy treatment. Scientists will initially only be able to correct enough mutations in cells to achieve a symptomatic cure. Though it may not be a complete cure for anemia, patients will be able to live a relatively normal life free of symptoms.
