Nucleic acids and methods of treatment for cystic fibrosis
AssigneeArcturus Therapeutics, Inc.
InventorsCarlos G. PEREZ-GARCIA, Kiyoshi TACHIKAWA, Daiki MATSUDA, Padmanabh CHIVUKULA, Priya Prakash KARMALI, Yanjie BAO, Jerel Boyd Lee VEGA, Rajesh MUKTHAVARAM, Amit SAGI
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Abstract
Nucleotides encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein are provided herein. Also describe are mRNA constructs that can be used to express CFTR protein in vitro or in vivo. The mRNA constructs can be formulated in a lipid formulation and administered via inhalation to treat cystic fibrosis.
Claims21
-86. (canceled)
A kit for expressing a human Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein in vivo, the kit comprising a 0.1 to 500 mg dose of a pharmaceutical composition comprising an mRNA comprising an open reading frame (ORF) having at least 80% sequence identity with one of SEQ ID NOs:100-105;
- and a device for administering the dose.
Dependencies are inferred from the claim text.