Gene therapy recent clinical applications

As we said in a previous article gene therapy is facing a new promising stage (see WHY). On 9 January this year, a review was published in JAMA on the latest advances in gene therapy, making reference to specific advances in humans. The first refers to gene therapy treatment of a...

CRISPR-Cas9, the gene editing tool, might cause cancer. Why does Crispr present difficulties in humans?

Editing cells’ genomes with CRISPR-Cas9  might increase the risk that the altered cells, intended to treat disease, will trigger cancer, two studies published on Monday warn [2018, Jun 11]— a potential game-changer for the companies developing CRISPR-based therapies. (Begley,...

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...

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...