Patent classifications
C12N9/6451
Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
This invention is directed to a vector which comprises a promoter operably linked to a nucleic acid sequence encoding the human C1 esterase inhibitor or Factor XII. The invention is also directed to a composition comprising the vector and a method of using the vector to treat or prevent hereditary angioedema.
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF COSMETIC PROTEINS AND PEPTIDES
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of cosmetic polynucleotides, cosmetic primary transcripts and cosmetic mmRNA molecules.
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF BIOLOGICS AND PROTEINS ASSOCIATED WITH HUMAN DISEASE
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF PROTEINS ASSOCIATED WITH HUMAN DISEASE
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
Miniature swine transgenic for one or more coagulation factors
Transgenic swine that express human coagulation factors, e.g., human coagulation factor VII, and/or one or more of human coagulation factors II, X and XII, and do not express the corresponding porcine coagulation factor or factors, as well as cells, tissues and organs derived therefrom, and their use in transplantation procedures.
NOVEL lNHIBITORS OF THE ENZYME ACTIVATED FACTOR XII (FXIIA)
The present invention relates to a bicyclic inhibitor of the coagulation enzyme activated factor XII (FXIIa) comprising or consisting of the peptide) (X.sup.1)(X.sup.2)(X.sup.3).sub.n(X.sup.4)RL(X.sup.5)(X.sup.6).sub.m(X.sup.7)(X.sup.9).sub.l(X.sup.10)(X.sup.11)(X.sup.12)(X.sup.13)(X.sup.14).sub.k(X.sup.15)(X.sup.16), wherein (X.sup.1) is present or absent and, if present, is an amino acid; (X.sup.2) is an amino acid with a side chain; (X.sup.3) is an amino acid and n is between 0 and 3, preferably 0 or 1 and most preferably 0; (X.sup.4) is an aliphatic L-amino acid or a cyclic L-amino acid, preferably L, P or an aromatic L-amino acid, and most preferably an aromatic L-amino acid; (X.sup.5) is an amino acid; (X.sup.6) is an amino acid and m is between 0 and 3, preferably 0 or 1 and most preferably 0; (X.sup.7) is an amino acid with a side chain; (X.sup.9) is an amino acid and l is between 0 and 3, preferably 0 or 1 and most preferably 0; (X.sup.10) is an amino acid; (X.sup.11) is an amino acid, preferably Q; (X.sup.12) is a hydrophobic L-amino acid, preferably an aliphatic L-amino acid, and is most preferably L; (X.sup.13) is an amino acid; (X.sup.14) is an amino acid and k is between 0 and 3, preferably 0 or 1 and most preferably 0, (X.sup.15) is an amino acid with a side chain; and (X.sup.16) is present or absent and, if present, is an amino acid; and wherein the side chains of (X.sup.2), (X.sup.7) and (X.sup.15) are connected via a connecting molecule, said connecting molecule having at least three functional groups, each functional group forming a covalent bond with one of the side chains of (X.sup.2), (X.sup.7) and (X.sup.15).
Modified polynucleotides for the production of cosmetic proteins and peptides
The invention relates to cosmetic mRNAs encoding elastin, and methods of using such mRNAs.
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF BIOLOGICS AND PROTEINS ASSOCIATED WITH HUMAN DISEASE
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF CYTOPLASMIC AND CYTOSKELETAL PROTEINS
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
IN VIVO PRODUCTION OF PROTEINS
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.