Nucleic acid products and methods of administration thereof
First Claim
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1. A method for treating a subject, comprising administering a synthetic RNA encoding a gene-editing protein targeting an immune checkpoint molecule gene, wherein the gene-editing protein comprises:
- (a) a DNA-binding domain comprising a plurality of repeat sequences and at least one of the repeat sequences comprises the amino acid sequence;
LTPvQWAlAwxyzGHGG (SEQ ID NO;
629) and is between 36 and 39 amino acids long, wherein;
“
v”
is Q, D or E,“
w”
is S or N,“
x”
is H, N, or I,“
y”
is D, A, I, N, G, H, K, S, or null, and“
z”
is GGKQALETVQRLLPVLCQD (SEQ ID NO;
630) or GGKQALETVQRLLPVLCQA (SEQ ID NO;
631); and
(b) a nuclease domain comprising a catalytic domain of a nuclease.
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Abstract
The present invention relates in part to nucleic acids, including nucleic acids encoding proteins, therapeutics and cosmetics comprising nucleic acids, methods for delivering nucleic acids to cells, tissues, organs, and patients, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, therapeutics, and cosmetics produced using these methods, kits, and devices.
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5 Claims
-
1. A method for treating a subject, comprising administering a synthetic RNA encoding a gene-editing protein targeting an immune checkpoint molecule gene, wherein the gene-editing protein comprises:
-
(a) a DNA-binding domain comprising a plurality of repeat sequences and at least one of the repeat sequences comprises the amino acid sequence;
LTPvQWAlAwxyzGHGG (SEQ ID NO;
629) and is between 36 and 39 amino acids long, wherein;“
v”
is Q, D or E,“
w”
is S or N,“
x”
is H, N, or I,“
y”
is D, A, I, N, G, H, K, S, or null, and“
z”
is GGKQALETVQRLLPVLCQD (SEQ ID NO;
630) or GGKQALETVQRLLPVLCQA (SEQ ID NO;
631); and(b) a nuclease domain comprising a catalytic domain of a nuclease. - View Dependent Claims (2)
-
-
3. A method for treating a subject comprising
(a) obtaining a cell comprising an immune checkpoint molecule gene and (b) contacting the cell with a synthetic RNA encoding a gene-editing protein capable of creating a single-strand or double-strand break in the immune checkpoint molecule gene, wherein the gene-editing protein comprises: -
(i) a DNA-binding domain comprising a plurality of repeat sequences and at least one of the repeat sequences comprises the amino acid sequence;
LTPvQVVAlAwxyzGHGG (SEQ ID NO;
629) and is between 36 and 39 amino acids long, wherein;“
v”
is Q, D or E,“
w”
is S or N,“
x”
is H, N, or I,“
y”
is D, A, I, N, G, H, K, S, or null, and“
z”
is GGKQALETVQRLLPVLCQD (SEQ ID NO;
630) or GGKQALETVQRLLPVLCQA (SEQ ID NO;
631); and(ii) a nuclease domain comprising a catalytic domain of a nuclease and wherein the synthetic RNA comprises one or more non-canonical nucleotides that avoid substantial cellular toxicity. - View Dependent Claims (4, 5)
-
Specification