Here is a paper from several years ago discussing a fascinating and very promising finding. We'll need to take a look at any subsequent developments in this field as they have occurred or will occur. Abstract:
We demonstrate editing of post-mitotic neurons in the adult mouse brain following injection of Cas9 ribonucleoprotein (RNP) complexes in the hippocampus, striatum and cortex. Engineered variants of Cas9 with multiple SV40 nuclear localization sequences enabled a tenfold increase in the efficiency of neuronal editing in vivo. These advances indicate the potential of genome editing in the brain to correct or inactivate the underlying genetic causes of neurological diseases.
The ability o directly alter gene sequences in the adult brain can yield a vast array of therapeutic options for certain genetic neurological diseases. An additional thing that will become necessary for certain of those diseases is the ability to therapeutically intervene, on brain-wide level, at the protein level as well (e.g., misfolded protein disorders).
No comments:
Post a Comment