Therapeutic gene editing for elane-associated disease
AssigneesChildren's Medical Center Corporation, University of Massachusetts
InventorsJosias Brito FRAZÃO, Scot A. WOLFE, Daniel E. BAUER, Shuquan RAO, Peter E. NEWBURGER
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Abstract
Provided herein are reagents and methods for targeting the ELANE gene for inhibition. Further provided herein is a method for producing a progenitor cell or a population of progenitor cells having decreased ELANE mRNA or protein expression.
Claims29
A modified synthetic nucleic acid comprising a nucleic acid sequence shown in Table 1, SEQ ID NOS:
- 1-374, wherein there is at least one chemical modification to a nucleotide in the nucleic acid molecule.
(canceled)
-10. (canceled)
-19. (canceled)
-26. (canceled)
-31. (canceled)
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-44. (canceled)
The method of claim 47, wherein the disease is selected from the group consisting of chronic obstructive pulmonary disease (COPD) (including acquired disease or genetic alpha-1 antitrypsin deficiency), cystic fibrosis, acute respiratory distress syndrome (ARDS), acute lung injury (ALI), asthmatic conditions, rheumatoid arthritis, chronic kidney disease, cyclic neutropenia, and severe congential neutropenia.
-51. (canceled)
Dependencies are inferred from the claim text.