New Gene Therapies?

Hox Genes

Certain genes that contain the homeobox sequence, the hox genes, are grouped in sequence along certain chromosomes. The order of this alignment reflects the positions of specific bones and body parts that these genes control.

Through the regulatory proteins that they produce, various groups of hox genes orchestrate the entire anatomical structure of the body, including the nervous system. The same genes in a different sequence create a different set of bones and other bodily parts.

GDF11 belongs to the transforming growth factor beta superfamily that controls anterior-posterior patterning by regulating the expression of Hox genes.

The precisely set circuits for biological regulation that genes help set in the “older parts” of the brain (brainstem and hypothalamus) comes to the rest of the brains as a more general arrangement of systems and circuitry.

This process goes on as a newly born develops and adapts itself when interacting with other individuals and its milieu. The genes that create the fins in fish are the same that create the 4 legs/arms in land creatures. Just different hox genes for on/off timing and level or intensity during the growth process. This started about 360 million years ago.

Thus there are coding genes that make the body parts (2%), non-coding switch genes that turn the former genes on and off, and non-coding control gene sequences that order the switch genes to go on or off. 

The fast majority of differences between the genes of animals vs. humans is in the switch-genes, not the coding genes.

The kind of response of the brain stem’s survival mechanism to each external event, strongly influences the strengthening or weakening of:

  • the patterns of connections among neurons,
  • their synapses, and thus
  • the neural development, resulting in
  • the kind of mindset the individual ends up with.

Gene Therapies

When people lack a certain enzyme because of defects in the gene that makes it, doctors can now inject a harmless virus that permanently implants a healthy working gene, thereby enabling patients to make that enzyme indefinitely.

Although still risky, trials have now reported successes against HIV, blood-clotting, and Parkinson’s disease.

A single fault in DNA then copied into RNA, produces the faulty proteins that underlie some genetic disorders.

Now biologists have designed ‘genomic debugger‘ proteins that bind to faulty RNA and fix protein production. 

The researchers realized that just 2 amino acids within the pattern of this designer protein determine exactly which base of an RNA molecule it will bind to.

Research results show that seeing specific number sequences will trigger action or in-action in certain genes in the DNA’s chromosomes of certain cells. 

New gene therapy delivery device could let hospitals create personalized nanomedicines on-demand. See below:

read more …

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