Duchenne muscular dystrophy could be treated with genome editing using CRISPR/Cas9 technique

Genome editing using  CRISPR/Cas9 is a promising possibility for correcting diseases due to genetic mutations (see HERE ), such as Duchenne muscular dystrophy – lethal degeneration of cardiac and skeletal muscle caused by different genetic mutations (see more HERE), which...

The slow advance of gene therapy now face a more promising chapter

Gene therapies are surely the most complex drugs in history. After a traditional trial-and-error phase, they now face a more promising chapter that is not without risks. Forty-five years ago in Science, Theodore Friedmann of the University of California, San Diego prophetically...

Therapeutic potential of Cas9-based epigenetic gene activation is proved in mice. VIDEO

 The Belmonte lab’s advanced in vivo Cas9-based epigenetic gene activation system in mice Salk scientists have created a new version technique of the CRISPR/Cas9 genome editing technology that allows them to activate genes without creating breaks in the DNA, potentially...

Mitochondrial transfer clinic opens its doors when several safety issues remain unclear

This tecnique include, in all its applications, generating of new individuals in vitro, who will only be implanted in their mothers if they are healthy and, if not, they will be discarded The first clinic offering mitochondrial transfer techniques, called Darwin Life, has been...

Open ethical debate: Are gene editing techniques ethical in reproductive medicine?

Author’s opinion: The use of these techniques is currently medically and ethically unjustifiable. The United Kingdom has recently approved mitochondrial transfer (3 parents children) to prevent the development of mitochondrial diseases in the children of mothers affected by...

First cell gene therapy to treat “Bubble boy” disease has been legalised in Europe

Last July, an article was published in medical journal Blood, which described the success of a cell gene therapy modality to treat the immunodeficiency caused by adenosine deaminase deficiency – “Bubble boy” disease. This is a disease that alters purine...

Mediterranean anaemia can be treated using gene therapy

Every year, around 300,000 children are born worldwide with β-thalassaemia, a type of hereditary anaemia that, that it presents asymptomatically in most cases, can become fatal. With around 250 million people affected, β-thalassaemia is estimated to be the most common hereditary...