Landmark CRISPR trial shows promise against deadly disease – Nature

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

Un comentario sobre “Landmark CRISPR trial shows promise against deadly disease – Nature

Agrega el tuyo

  1. Reblogueó esto en Pozos de Pasióny comentado:

    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.

Deja un comentario

Create a website or blog at WordPress.com Tema: Baskerville 2 por Anders Noren.

Subir ↑

A %d blogueros les gusta esto: