Medical Technology Stock Letter

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« The Human Genome Project Part 3: Stem Cells | Main | Did you short Vivus (VVUS) before the FDA Panel Meeting? »

The Human Genome Project: Part 2

Whereas gene therapy was once the province of scary stories – about parents engineering their perfect children or laboratory created abominations – the technology has now progressed to the point that meaningful clinical trials are being attempted.  When talking about gene therapy in medicinal terms, we are usually talking about somatic gene therapy, meaning therapy for the living bodily, or somatic cells.  This is opposed to germ line therapy, which involves changing the DNA of sperm or egg cells to (possibly) alter the traits of offspring.  Unlike somatic therapy, changes in the germ line DNA will be passed from generation to generation.  Explaining the difference between germ-line and somatic gene therapy will be important for the industry as it will allay the worst fears of uncontrolled genetic changes replicating throughout the environment...

The Gene Desert
As if deciphering the gene-coding region of the human genome wasn’t enough work, genomic studies are finally giving more insight into the vast majority of DNA that doesn’t contain genes.  This area – junk DNA or the gene desert – no doubt contains more useful information than is yet realized.  A large portion, it turns out, transcribes many different types of RNA, which – we are discovering – play a much larger role in the cellular machinery than was imagined.  Other portions contain information telling genes when to turn off or on, or regulate cells under sub-optimal conditions.  Although not all biologists have come around to this view, we firmly believe that there is far too much evolutionary pressure on DNA for any more than a tiny fraction to actually be ‘junk’.

 
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