Methods and products for expressing proteins in cells
AssigneeFactor Bioscience Inc.
InventorsMatthew Angel, Christopher Rohde
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Abstract
The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Claims9
An in vitro method for editing a nucleic acid sequence in a genome of a cell comprising a Duchenne muscular dystrophy (DMD) gene, comprising transfecting in vitro the cell with a synthetic ribonucleic acid encoding a gene-editing protein,
- wherein the gene-editing protein targets a nucleic acid sequence within the DMD gene and within about 1 kilobase (kb) of a splice acceptor site upstream of an exon to be skipped and
- the gene-editing protein comprises:
- (a) a DNA-binding domain and (b) a nuclease domain, wherein:
- (a) the DNA-binding domain comprises a plurality of repeat sequences and at least one of the repeat sequences comprises the amino acid sequence:
- LTPvQWAlAwxyzGHGG (SEQ ID NO:
- 75) and is between 36 and 39 amino acids long, wherein:
- “v” is Q, D or E,
- “w” is S or N,
- “x” is N or H,
- “y” is D, A, H, N, K, or G, and
- “z” is GGKQALETVQRLLPVLCQD (SEQ ID NO:
- 670) or GGKQALETVQRLLPVLCQA (SEQ ID NO:
- 671);
- and
- (b) the nuclease domain comprises a catalytic domain of a nuclease,
- to result in editing of the nucleic acid sequence in the cell.
Dependencies are inferred from the claim text.