Analysing the data recently presented by Jiankui He and assuming that it is authentic shows that the goal of abolishing the expression of CCR5 may have been reached for one of the resulting twins, although this remains to ...

Viable human embryos carrying a disease mutation have now been corrected by CRISPR-Cas9 editing. Although the correction is not perfect, and the embryos have not been implanted, this brings us closer to human germline ...

In vivo gene editing has been achieved in a phase I clinical trial and results in a strong reduction of the level of a pathogenic protein. While preliminary, these results open the way for many ...

Two clinical trials using CRISPR technology to engineer the T cells of cancer patients by inactivating the gene for immune checkpoint molecule PD-1 are (almost) underway, one in the USA and the other in China. The less ...

A new approach, based on the use of peptide nucleic acid molecules to form triple helices and promote locus-specific recombination, demonstrates potential for in vivo gene correction at clinically significant levels and ...

Une preuve de concept (en anglais POC ou Proof of concept) est la validation expérimentale d’une hypothèse de travail afin d’en démontrer le bien-fondé. Dans le contexte qui nous occupe ici, il s’agit de la démonstration ...

Les dystrophies musculaires sont un ensemble de pathologies musculaires rares, caractérisées par une faiblesse et une dégénérescence progressive du muscle. Ce sont des maladies d’origine génétique causées par la mutation ...

Diseases of the locomotor system are at the origin of disabilities with severe social and economic consequences. The study of the neuromuscular system development and maintenance has become a key challenge for the scientific ...