CFTR MRNA compositions and related methods and uses
AssigneesTranslate Bio, Inc., ethris GmbH
InventorsMichael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
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Abstract
The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.
Claims13
A non-naturally occurring mRNA molecule comprising a coding sequence, a 5′-untranslated region, (5′-UTR), and a 3′-untranslated region, (3′-UTR), wherein the coding sequence encodes the amino acid sequence of SEQ ID NO:
- 1 and the coding sequence is at least 80% identical to SEQ ID NO:
- 3.
Dependencies are inferred from the claim text.