Gene and RNA supplementation in post-mitotic cells in monogenic recessive disorders using mRNA or recombinant adeno-associated viruses (rAAV) for gene transfer
For diseases or syndromes with recessive, monogenic inheritance, supplementation of the correct/wild-type version of the gene by transfer of intact mRNAs or cDNAs into target cells, which complements the defective gene, enters the nucleus and is transcribed to mRNA ibidem; then, correct proteins are translated from this mRNA, which complements the endogenous mutated proteins or substitutes missing proteins. This strategy provides a causal therapy, which is supposed to last lifelong after being applied only once.
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