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Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith: CFTR mRNA compositions and related methods and uses. Shire Human Genetic Therapies, Ethris, Choate Hall & Stewart, Fangli Chen, November 10, 2015: US09181321 (2 worldwide citation)

Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.


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Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith: CFTR mRNA compositions and related methods and uses. RaNA Therapeutics, Ethris, Fangli Chen, Proskauer Rose, July 25, 2017: US09713626

Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising th ...