Se acerca el deseado, y temido, momento en el que sea posible curar enfermedades incurables agazapadas en nuestro genoma. Será más dura la labor social de divulgación y eliminación de temores arraigados en creencias y morales religiosas que el desarrollo de la técnica, estoy segura. Aunque también estoy segura de que las personas cuyas vidas se puedan salvar serán incontables y pesarán en la balanza.
Administering gene-editing treatment directly into the body could be a safe and effective way to treat a rare, life-threatening condition.
Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene-editing can be deployed directly into the body to treat disease. The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components — in this case targeting a protein that is made mainly in the liver — are infused into the bloodstream. In the trial, six people with a rare and fatal condition called transthyretin amyloidosis received a single treatment with the gene-editing therapy. All experienced a drop in the level of a misshapen protein associated with the disease. Those who received the higher of two doses tested saw levels of the protein, called TTR, decline by an average of 87%.
Origen: Landmark CRISPR trial shows promise against deadly disease
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