How did mRNA technology contribute to evolution. Which diseases could be addressed using this technique.
Scientists from the USA announced that they have developed a method to directly reprogram bone marrow cells within the body.
If this technique proves equally successful in clinical practice, it could potentially replace hematopoietic stem cell transplants in the future. These are performed on patients with hematological disorders (e.g., leukemia) after undergoing intensive chemotherapy.
Furthermore, it may also lead to the treatment of previously incurable diseases, such as hemoglobinopathies (e.g., sickle cell anemia).
The method is based on the direct delivery of mRNA into a patient’s bone marrow stem cells. This is achieved using a technique similar to the one developed for coronavirus vaccines.
Once the mRNA reaches the target cells, it corrects the genetic mutations responsible for the specific disorder. As a result, the bone marrow of the patient begins to produce healthy cells.
Scientists from the Children’s Hospital of Philadelphia (CHOP), who developed the method, successfully applied it in experiments on animals and in cellular series in the laboratory.
They corrected a genetic mutation.
The new findings are being published in the scientific journal Science. As explained by the researchers, they tested their technique on the bone marrow of living mice and on hematopoietic stem cells from four patients with sickle cell disease.
In human samples, the method corrected the genetic mutation that causes a portion of the patients’ red blood cells to have a sickle shape. The normal shape of red blood cells is oval.
This discovery suggests that gene editing of bone marrow could be feasible without the usual process used today.
The typical procedure involves finding a compatible donor and obtaining hematopoietic cells from them. These cells are then transplanted into the patient, who must take medication for a significant period to prevent rejection by the body.
The new findings could potentially revolutionize genetic therapies, stated Dr. Laura Breda, Head Researcher and Associate Professor of Hematology at CHOP.
For instance, they could lead to the treatment of both hematological and non-hematological disorders caused by specific genetic mutations, such as:
- Hemoglobinopathies (e.g., sickle cell anemia, thalassemia)
- Inherited anemias or thrombocytopenias
- Immune deficiencies
- Cystic fibrosis
- Various metabolic disorders
- Muscular dystrophies
All of these conditions could potentially be addressed through a simple intravenous infusion of targeted gene therapies,” she said. However, she was quick to clarify that this won’t happen in the near future. Many more research efforts are needed before the method can be tested in humans, she emphasized.