Cellscript, LLC

University of Pennsylvania

Other

Bio-techne Corporation

Anthony Person

Gary A Dahl

Jerome J Jendrisak

Judith Meis

Drew Weissman

Katalin Kariko

Cynthia Chin

Katalin Karikó

Liwen Xiong

Ming Bi

Camrin Tracy

The present invention relates to compositions, kits and methods for making and using RNA compositions comprising in vitro-synthesized ssRNA inducing a biological or biochemical effect in a mammalian cell or organism into which the RNA composition is repeatedly or continuously introduced. In certain embodiments, the invention provides compositions and methods for changing the state of differentiation or phenotype of a human or other vertebrate cell. For example, the present invention provides mRNA and methods for reprogramming cells that exhibit a first differentiated state or phenotype to cells that exhibit a second differentiated state or phenotype, such as to reprogram human somatic cells to pluripotent stem cells.

Making and using in vitro-synthesized ssRNA for introducing into mammalian cells to induce a biological or biochemical effect

The present invention provides compositions and methods for reprogramming somatic cells using purified RNA preparations comprising single-strand mRNA encoding an iPS cell induction factor. The purified RNA preparations are preferably substantially free of RNA contaminant molecules that: i) would activate an immune response in the somatic cells, ii) would decrease expression of the single-stranded mRNA in the somatic cells, and/or iii) active RNA sensors in the somatic cells. In certain embodiments, the purified RNA preparations are substantially free of partial mRNAs, double-stranded RNAs, un-capped RNA molecules, and/or single-stranded run-on mRNAs.

RNA preparations comprising purified modified RNA for reprogramming cells

This disclosure describes isolated protein complexes including a Wnt and a sFRP; compositions including a Wnt; a cell overexpressing a Wnt and a sFRP; compositions including a cell overexpressing a Wnt and a cell overexpressing a sFRP; methods of making the protein complexes, compositions, and cells; and methods of using the isolated protein complexes, compositions, and cells. This disclosure further describes methods of forming a complex including a Wnt and a sFRP and methods for isolating a Wnt. Also described herein are methods that may be used to purify a Wnt without the use of a detergent.

Wnt/sFRP complexes, Wnt-containing compositions, Wnt-expressing cells, and methods of making, purifying, and using same

The present invention provides methods, kits, and compositions for reducing an innate immune system response in a human or animal cell, tissue or organism. One embodiment comprises: introducing an Agent mRNA comprising in vitro-synthesized mRNA encoding one or more proteins that affect the induction, activity or response of an innate immune response pathway; whereby, the innate immune response in the cell, tissue or organism is reduced compared to the innate immune response in the absence of the Agent mRNA. Other embodiments are methods, compositions and kits for using an Agent mRNA for treating a disease or medical condition in a human or animal that exhibits symptoms of an elevated innate immune system, or for reducing an innate immune response that is induced in a human or animal cell, tissue or organism by a Foreign Substance that is administered to the cell, tissue or organism.

Inhibition of innate immune response

The present invention relates to methods for changing the state of differentiation of a eukaryotic cell, the methods comprising introducing mRNA encoding one or more reprogramming factors into a cell and maintaining the cell under conditions wherein the cell is viable and the mRNA that is introduced into the cell is expressed in sufficient amount and for sufficient time to generate a cell that exhibits a changed state of differentiation compared to the cell into which the mRNA was introduced, and compositions therefor. For example, the present invention provides mRNA molecules and methods for their use to reprogram human somatic cells into pluripotent stem cells.

Compositions and methods for reprogramming eukaryotic cells

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Madison, WI, United States of America

Anthony Person