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Gene Replacement mRNA

Many genetic disorders are recessive. In many cases, gene replacement has potential to ameliorate recessive disorders by replacing the defective protein. Historically, DNA based non-viral and viral vector based approaches have been used for gene replacement. However, concerns about insertional mutagenesis have been raised about these approaches. More recently, mRNA transfection has gained popularity for several reasons. First, with mRNA transfection, there is no risk of insertional mutagenesis. Second, in contrast to plasmid and viral vector based approaches which must reach the nucleus before gene expression, mRNAs only need to cross one membrane to be expressed in the cytoplasm. This may reduce the delivery hurdles that must be overcome before gene replacement can become a reality in the clinic. TriLink offers a series of well established gene replacement markers that can be used to assess efficacy of mRNA delivery.

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CleanCap Erythropoietin (EPO) mRNA (5moU)

 

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