Gene therapy gets a new boost.

Gene therapy real drug will now begin to translate beyond clinical trials Gene therapy has received a new boost this year with the approval of Kymriah from Novartis by the United States regulatory agency, the FDA. The treatment is indicated for acute lymphoblastic leukemia in...

Can one gene link two unrelated diseases?

Every genetic disease is traditionally associated with one gene bit now a study has been published suggesting that one gene links unrelated diseases playing a role in Parkinson’s disease and Crohn’s disease, a serious bowel disorder. The team found a link between Crohn’s disease...

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...

The embryo germline modification, particualrly in humans. It is an unsafe procedure

 “Absolute prohibition of such experiments” A group of clinical researchers has proposed modifying embryos using “therapies that may affect the embryo germline“. These techniques could produce changes that could be transmitted to successive generations....

Ethical questions about mitochondrial replacement in humans. Three-parent baby technique

We thus consider it necessary to establish a moratorium on their use in humans, at least until more is known about these aspects. If this knowledge is obtained, ethical questions would still remain to be resolved, among which we consider the most relevant to be those related to...

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...
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