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
Life of this patent
Estimating the term…
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.
Claims89
An mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA comprises an open reading frame (ORF) having at least 80% sequence identity with one of SEQ ID NOs:
- 100-105.
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
(canceled)
Dependencies are inferred from the claim text.