Anavex Life Sciences Corp (AVXL)

[falconer66a]

But, Has It Worked? Can It Work?

Thanks for the more expansive perspective of CRISPR technology.

Might it, eventually, be able to be administered externally to provide systemic relief of a general central nervous system genetic defect in a human?

Are you speaking conceptually, or are there any actual, model cases of this that would provide a model and example of useful genetic manipulation therapy for genetically-caused Alzheimer's or Parkinson's?

Tell us. Has it worked yet. I'm not aware of any such outcomes. Getting the CRISPR protein across the blood-brain barrier, then across the plasma membrane, into a functioning nerve cell, appears to be a daunting task.

Explain, please, the biophysics of the "nanoparticles" that seem essential for all of this to work. How can they get into the cell; then, how do they unwind the protective chromosomal chromatin surrounding DNA to gain reactive access to the then naked nucleotide sequences? All of that can be done in lab cultures of pure cells. Has it yet been done in systemic human cells for anything like Alzheimer's? If not, when? (And, how?)

And, of course, only a fraction of Alzheimer's cases are caused by a single gene defect amenable to CRISPR tech. The specific genotypic pathology of most Alzheimer's cases is utterly idiopathic, of unknown cause, genetic or otherwise. For CRISPR technology to work, all controlling genes must be known and targeted. With all of the genomic analysis of Alzheimer's genomes, there has been scant discovery of causative genes, beyond ApoE. Correct?