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Replies to #7184 on Biotech Values
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aslan2772

02/01/05 12:48 AM

#7213 RE: DewDiligence #7184

If I understand TrpRS therapy correctly, they will want to increase levels of the protein in the back of the eye. This cannot be accomplished with RNA-interfereing morpholinos or any other known oligo nucleic acid-based (or their derivatives) treatment --current RNAi treatments that I am aware of only decrease expression of the target gene product. The TrpRS strategy would seem to require that either the protein itself be delivered (injected, eluted from a device or from heterologous cells which express and secrete the protein), or alternatively, infection/transfection the back of the eye with a fully encoding DNA sequence (aka Gene therapy).

A missing piece of this puzzle is the cellular location of the target of TrpRS (intracellular or extracellular???). While small nucleotide sequences (oligos) can be modified to cross the cell membrane, usually neither genes nor the proteins they encode will cross the cell membrane to any appreciable extent without some sophisticated trickery. Now, if they can identify and clone the "receptor" for TrpRS, they might as well just design/find a drug for it!