Gene editing and stem-cells therapy to treat blood diseases

Researchers from Boston and Shanghai have recently published an interesting article in Nature medicine on their research using gene editing in human stem cells, with the aim of advancing the development of therapeutic strategies to treat different blood diseases.

Change of sequence v. change a single letter of the genome

In the prolific field of gene editing, numerous advances and strategies are developing rapidly. Among them is so-called base editing, which consists of changing a single letter of the genome. This is a much more delicate and precise approach than the change of sequences made in standard gene editing, resulting in a reduction in the risk of unwanted alterations in the genome.

gene editing and stem-cells gene editing innovation

New innovative gene therapy technique

Gene editing innovation to treat Cell anemia and β-thalassemia • diseases

This is the strategy that researchers in the aforementioned study have applied to human hematopoietic stem cells, precursors of blood cells and the immune system. The results published in the journal demonstrate the successful editing of different gene sites involved in hematological diseases such as sickle cell anemia and β-thalassemia.

From a bioethical standpoint, research with these types of stem cells is not only acceptable but desirable, since these are adult stem cells. Thus, the destruction of human embryos is not required (as is the case with embryonic stem cells) and could lead to positive medical advances that may otherwise be unattainable.

This technique gene editing and stem-cells therapy still remain unsafe

Nevertheless, the application of gene editing and stem-cells therapies in clinical practice still requires the resolution of the remaining safety problems: off-target effects, i.e. genome modification not only at the desired site but also elsewhere, which can be very risky. In the paper discussed, the researchers detected different off-target genetic modification events, and therefore recommend that more comprehensive studies be conducted before they can be used in human medicine.

  • Thalassemia (thal-uh-SEE-me-uh) is an inherited blood disorder that causes your body to have less hemoglobin than normal. Hemoglobin enables red blood cells to carry oxygen. Thalassemia can cause anemia, leaving you fatigued.