Anavex 2-73 Won’t Be “Gene Therapy;” Better
“Gene therapy” is generally regarded as the manipulation of nucleotide sequences, the deliberate changing of a gene’s genetic code. The code’s letters are Adenine = A, Thymine = T, Cystine = C, and Guanine = G.
A’s always (except in a mutation) connect to a T. C’s to G’s.
In gene therapy, now using CRISPR technology, new, improved, or fixed genes are inserted into the DNA of chromosomes, where the new nucleotide sequences, the chemical genes, code for proper peptides. Peptides are short, unfolded amino acid sequences. In the endoplasmic reticulum, properly-sequenced peptides are connected, making real proteins.
But in most cases, those proteins then have to be exquisitely folded into precise, complicated shapes to be functional. In most cases, the result is a functioning protein enzyme, which goes on to mediate and control virtually all of the cell’s chemistry.
First, sequence the amino acids properly, the function of the DNA code. Then, connect those amino acids in sequence. This happens in the ribosome. The connected amino acids are the peptides, which then get connected and properly bent into shape in the endoplasmic reticulum (where the Anavex sigma-1 receptor agonists have their function).
Now, back to Anavex 2-73. It does none of this, per se. Therefore, it is not “gene therapy.”
But in most cases (even better than gene therapy), it simply (well, rather “complicatedly”) causes all of the described processes to occur normally; particularly proper protein folding. Good enzymes are then produced. The cell must necessarily function normally.
Gene therapy is controversial. Messing with, changing DNA and genes, has a host of ethical issues. CRISPR-based gene therapies fall under a pile of regulations, restrictions, and prohibitions (as with the Chinese researcher who performed CRISPR technology on an in utero human fetus. He got fired.)
Anavex will encounter none of that. It doesn’t change genes. It facilitates their proper, healthful functions. In the infrequent cases of genetic diseases (as with Rett syndrome), Anavex 2-73 will not create a normal, healthful genotype. The little girls will continue have malfunctioning genes. But Anavex 2-73 promotes normal non-genetic functions that can suppress the untoward outcomes of the bad genes (as demonstrated in Rett-type mice; and soon enough, in real humans).
Anavex therapies will not be complicated or compromised by the restrictions and prohibitions of regulated gene therapy.
