Significats de directed gene en anglès

However, airway- directed gene delivery is not as simple as was originally anticipated.

These data suggest that CNS- directed gene therapy synergizes with BMT.

These results may have potential applications in the development of muscle- directed gene and cell therapies.

Moreover, the first two technologies should be generalizable to all liver- directed gene therapy vector designs.

In the last two decades, muscle- directed gene therapies were primarily focused on treating recessive disorders.

Liver- directed gene therapy hopes to achieve long-term correction of the disease while also inducing immune tolerance.

Our findings demonstrate the applicability of nuclease-loaded 'all-in-one' IDLVs for site- directed gene insertion in stem cell-based gene therapies.

Factors relevant to the successful application of adeno-associated virus (AAV) vectors for liver- directed gene therapy were evaluated.

The nucleolytic activity of animal Argonaute proteins is deeply conserved, despite its having no obvious role in microRNA- directed gene regulation.

This study is the first to demonstrate long-term safety and efficacy of hepatocyte- directed gene therapy in a large animal model.

Here we take advantage of newly available molecular genetics for Cryptosporidium parvum to investigate nucleotide biosynthesis by directed gene ablation.

The data indicate that adenovirus-mediated delivery of Bcl-2 resulted in a significant increase in NFkappaB-dependent DNA binding and NFkappaB- directed gene transcription.

In addition, the therapeutic efficacy may have been limited since the CNS- directed gene therapy was restricted to the forebrain and thalamus.

However, conventional techniques of directed gene expression or ablation confound adult heart phenotypes with genetic perturbation of embryonic or postnatal cardiac development.

In contrast, vector-based, cardiomyocyte- directed gene therapy using murine and human H19 strongly attenuated heart failure even when cardiac hypertrophy was already established.

These data are truly striking, given that BMT alone is ineffective, yet it synergizes with CNS- directed gene therapy to dramatically increase efficacy and lifespan.