This review attempts to survey the potential to use traditional pharmaceuticals as opposed to biopharmaceuticals to treat single gene disorders. The available strategies include the use of antisense oligonucleotides to alter splicing or knock-down expression of a transcript, siRNAs to knock-down gene expression, and drugs for nonsense mutation read-through.
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This review attempts to survey the potential to use traditional pharmaceuticals as opposed to biopharmaceuticals to treat single gene disorders. The available strategies include the use of antisense oligonucleotides to alter splicing or knock-down expression of a transcript, siRNAs to knock-down gene expression, and drugs for nonsense mutation read-through.