摘要:
The present invention relates to a Crumbs homologue (CRB) therapeutic for use as a medicament or in a method of treatment or prophylaxis, for example in the treatment or prophylaxis of a retinal disorder due to mutations in the Crumbs homologue-1 (CRB1) gene, such as Leber's congenital amaurosis 8 (LCA8) or retinitis pigmentosa 12 (RP12). In particular, the present invention relates to a recombinant viral vector comprising CRB2 or modified non-toxic forms of either CRB1 or CRB3 that resemble CRB2.
摘要:
The present invention relates to a method for detecting cell death using a luminescent compound; to the luminescent compounds for particular uses; to a kit comprising compounds and to a protein. The method is applicable for detecting cell death, essentially regardless of the mechanism through which cell death occurred or is occurring and is therefore not limited e.g. to detecting cell death resulting from only one mechanism selected from apoptosis and necrosis.
摘要:
The present invention provides a reverse genetics system for viruses belonging to the Reoviridae (i.e. Reoviruses), various uses thereof, genetically modified Reoviruses, Reovirus selection/production and propagation systems, medicaments and vaccines.
摘要:
Novel nucleic acid sequences, vectors, modified cells, peptides and pharmaceutical compositions are provided that are useful in the treatment of human subjects having a ΔNPM 1 positive haematological malignancy. Corresponding methods and uses are also provided.
摘要:
The technology relates in part to compositions and methods for inducing an immune response against the Preferentially Expressed Antigen of Melanoma (FRAME). Provided are methods for treating hyperproliferative diseases by inducing an immune response against PRAME antigen; the immune response may be induced by specifically targeting PRAME-expressing cells using T cell receptors directed against PRAME.
摘要:
Disclosed is a method and composition for treating tumors or infectious diseases, wherein the composition comprises an agonistic anti-CD40 antibody or a fragment thereof, and/or an agonistic anti-4-1BB antibody or a fragment thereof. The agonistic antibody molecules include fragments like Fab, (Fab')2 and Fv. The methods and compositions may further comprise polypeptides or mucleic acid sequences having or encoding a class 1 MHC or class II MHC-restricted T cell epitope.
摘要:
Novel nucleic acid sequences, vectors, modified cells, peptides and pharmaceutical compositions are provided that are useful in the treatment of human subjects having a ΔNPM 1 positive haematological malignancy. Corresponding methods and uses are also provided.
摘要:
The invention among other provides means and methods for modulating NOTCH3 expression and/or protein coding domain. In one aspect the invention provides a method for at least reducing an elevated level of NOTCH3 protein in a NOTCH3 expressing cell or the immediate vicinity thereof said method comprising providing said cell with an anti-sense oligonucleotide specific for NOTCH3 m RNA or pre-m RNA thereby decreasing production of said NOTCH3 protein or thereby altering the protein coding region in said NOTCH3 m RNA or pre-m RNA.