The present disclosure relates to RNA interference (RNAi) reagents for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.

The present invention relates to a polypeptide or protein for use in a method of diagnosis or treatment of an autoimmune disease in a subject, characterized in that said polypeptide or protein comprises one or more epitopes derived from the protein DPPX. Further, the invention relates to a nucleic acid or vector encoding such polypeptide, to a cell comprising such a vector, to an in vitro diagnostic method and test kit involving such polypeptide, to a pharmaceutical composition comprising such polypeptide, to a medical device coated with such polypeptide or pharmaceutical composition and to methods for treating an autoimmune disease in a subject.

Peptides that specifically bind erythrocytes are described. These are provided as peptidic ligands having sequences that specifically bind, or as antibodies or fragments thereof that provide specific binding, to erythrocytes. The peptides may be prepared as molecular fusions with therapeutic agents, tolerizing antigens, or targeting peptides. Immunotolerance may be created by use of the fusions and choice of an antigen on a substance for which tolerance is desired.

Peptides that specifically bind erythrocytes are described. These are provided as peptidic ligands having sequences that specifically bind, or as antibodies or fragments thereof that provide specific binding, to erythrocytes. The peptides may be prepared as molecular fusions with therapeutic agents, tolerizing antigens, or targeting peptides. Immunotolerance may be created by use of the fusions and choice of an antigen on a substance for which tolerance is desired.

Described is a construct comprising (a) a targeting moiety; (b) a fusogenic moiety consisting one or more fusogenic sequence(s) derived from dengue virus glycoprotein E comprising the sequence DRGWGNGCGLFGKGGI (SEQ ID NO:1) or a sequence which shows 1 to 8 substitutions, deletions, or insertions in comparison to SEQ ID NO:1; and (c) a molecule which is to be delivered into the cytoplasm of a cell. Moreover, described is a pharmaceutical composition comprising the construct according to the invention and optionally a pharmaceutical acceptable carrier. Further, described is a kit comprising one or more fusogenic sequence(s) derived from dengue virus glycoprotein E comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 8 substitutions, deletions, or insertions in comparison to SEQ ID NO:1. Further, described is the use of one or more fusogenic sequence(s) derived from dengue virus glycoprotein E for use in delivery of a therapeutic moiety, a detectable moiety, a nucleic acid molecule, preferably an siRNA, a carrier molecule, preferably a nanoparticle, a liposome and a viral vector into the cytoplasm of a cell.

The present disclosure provides methods for generating Otub 1 deficient T cells and natural killer (NK) cells and compositions comprising engineered T cells expressing a reduced amount of Otub 1. Further provided are methods of treating cancer comprising administering the Otub 1 deficient T cells and/or NK cells to a subject in need thereof.

Proteinaceous heterodimers, pharmaceutical compositions, medicaments and/or kits comprising the proteinaceous heterodimers, methods for producing the proteinaceous heterodimers, and uses thereof.

The present disclosure relates generally to peptide reporter constructs of SNAP-25, which are useful in determining the activity of botulinum toxins, and methods of using the same.

Provided herein are methods for preparing and characterizing SARS-co2 antigen specific immune cell cultures and preparations and methods of using the same in adoptive immunotherapy for cancer, infections and immune disorders. Also, provided are compositions and methods for generating immune cells expressing synthetic antigen binding receptors targeting SARS-cov2 and methods of use of these cells for the treatment and prevention of COVID-19. Also provided are compositions and methods for determining immune response to SARs-cov2 in a subject, detecting SARS-cov2, measuring cytotoxicity induced by SARS-cov2, and detecting the expression and cytotoxicity of synthetic antigen binding receptors targeting SARS-cov2.

The present disclosure relates to a tyrosine hydroxylase (TH) variant lacking 60 to 120 amino acid residues at the N terminus, and a pharmaceutical composition comprising the TH variant lacking 60 to 120 amino acid residues at the N terminus and the aromatic L-amino acid decarboxylase (AADC). The present disclosure further relates to a nucleotide construct, a vector plasmid, a cell or a virus comprising the TH variant or the pharmaceutical composition, as well as use of the virus in the manufacture of a medicament for treating neurodegenerative diseases (e.g., Parkinson's Disease).