Patent ID: 11951184
Assignee: WISCONSIN ALUMNI RESEARCH FOUNDATION
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C  Y | IPC A  C

Claim 0:
1. An expression cassette for gene silencing and replacement of a target protein, comprising, in operable communication,
a promoter,
an expression attenuator, wherein the expression attenuator comprises one or more 5′UTR hairpins, a pseudoknot, a viral or cellular internal ribosomal entry site (IRES), an RNA G-quadruplexe (RG4s), an upstream open reading frames (uORF), or an upstream start codon (uAUG),
a nucleotide sequence encoding a gene for the replacement target protein, and
an shRNA sequence for knockdown of an endogenous variant of the target protein,
wherein the promoter, the expression attenuator, the nucleotide sequence encoding the gene for the replacement target protein, and the shRNA are expressed as a single transcript.