progress for children?

CRISPR technology, which allows genes to be manipulated with unparalleled precision, is taking a new step: the first clinical tests to treat two genetic diseases will be extended to children under 12.

Already, the first trials among 12 to 35 year olds to treatanemia (Anemia (from the privative an- and from the ancient Greek haimos, “blood”) is a…) sickle-shaped (or sickle cell disease (Drepanocytosis (from the Greek drepnos, sickle), also called hemoglobinosis…), one of the most common genetic diseases) and beta-thalassemia, were described as promising. Both are genetic abnormalities of hemoglobin that result in inadequate, or insufficient, production. Symptoms are numerous and include anemia and increased susceptibility to infections. Some can be treated with a bone marrow transplant (Marrow transplantation is a transplant involving the transfer of bone marrow…) bone.

In theory (The word theory comes from the Greek word theorein, which means “to contemplate, to observe,…), altering the offending genes would be the solution, an intervention that only became possible when CRISPR became a reality and we saw, in the 2010s, an accumulation of breakthroughs in animals. In this case, it is a question of replacing, at the patient (In the field of medicine, the term patient commonly refers to a person receiving…)his “defective” blood cells by his own “genetically altered” blood cells.

For the first time, we therefore envisage being able to modify these genes in children, and potentially have an impact on their development. However, there is still a long way to go: the “promising” results of clinical trials on 12-35 year olds, unveiled on June 12, are only those said to be phase (The word phase can have several meanings, it is used in several fields and…) 2 – on a limited group of people, in this case 75 people. The everything (The whole understood as the whole of what exists is often interpreted as the world or…) first trials started in 2019.

If the promising results are confirmed, it will therefore be years before we hope to see the treatment used in large scale (The large ladder, also called aerial ladder or auto ladder, is a…)and that’s not counting the Side effects still poorly understood, the specialized personnel that will have to be trained.

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