Provided herein are genetically engineered gas vesicle expression systems (GVES) that are configured to express gas vesicles (GVs) in a mammalian cell, related gas vesicle polynucleotide constructs, gas vesicle reporting genetic circuits, vectors, genetically engineered mammalian cells, non-human mammalian hosts, compositions, methods and systems, which in several embodiments can be used together with contrast-enhanced imaging techniques to detect and report biological events in an imaging target site comprising a mammalian cell and/or organism.

Hepatitis B Virus (HBV) antigen specific binding molecules, in particular T Cell Receptors (TCRs), TCR polypeptides and fragments thereof. The invention is also related to modified cells containing the TCRs, TCR polypeptides or fragments, pharmaceutical composition or kits including the same or methods of making or using the same as is described. In particular, the invention discloses TCRs or a fragments thereof, capable of binding to a peptide of a Hepatitis B Virus (HBV) Env polypeptide presented by an MHC class I molecule comprising an MHC class I σ-chain encoded by an HLA-Cw*08 allele.

There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional α-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.

Provided herein are new compositions and methods to target pharmaceutical agents to pathological areas by utilizing fusion polypeptides. These fusion polypeptides contain two or more domains: (i) aptamer sequences that bind to exposed collagenous (XC-) proteins present in pathological areas, including cancerous and viral lesions, (ii) immunomodulators, such as cytokines, and optionally (iii) at least one linker joining the two domains or at the terminus of the polypeptide. In some cases, the linker is a rigid linker, e.g., a rigid helical linker. Also provided herein are methods of treating cancer and/or infectious diseases using the new fusion polypeptides.

IPEX (Immune dysregulation Polyendocrinopathy X linked) syndrome is a primary immunodeficiency caused by mutations in the gene encoding the transcription factor forkhead box P3 (FOXP3), which leads to the loss of function of thymus-derived CD4+CD25+ regulatory T (tTreg) cells. Preclinical and clinical studies suggest that T cell gene therapy approaches designed to selectively restore the repertoire of Treg cells by transfer of wild type FOXP3 gene is a promising potential cure for IPEX. However, there is still a need for a vector that can be used efficiently for the preparation of said Treg cells. The inventors thus compared 6 different lentiviral constructs according to 4 criteria (vector titers, level of transduction of human CD4+ T cells, level of expression of FOXP3 and ΔLNGFR genes, degree of correlation between both expression) and selected one construct comprising a bidirectional PGK-EF1a promoter that showed remarkable efficiency.

The disclosure relates generally to bacteria that have been modified to have increased oxalate degrading activity, pharmaceutical compositions including the bacteria, and methods of treating disorders associated with an elevated amount of oxalate, e.g., hyperoxaluria.

A method for producing a recombinant adeno-associated virus (rAAV) and a recombinant baculovirus virus, the method including: (1) infecting an insect host packaging cell line with a corresponding recombinant baculovirus integrated with an rAAV genome ITR-GOI (gene of interest flanked by AAV inverted terminal repeats) and an AAV Cap gene or AAV Rep gene; (2) culturing the host packaging cell line infected with the recombinant baculovirus, so as to produce the recombinant adeno-associated virus; and (3) separating and purifying the recombinant adeno-associated virus obtained in (2).

A nucleic acid comprising a first nucleic acid region comprising a nucleic acid sequence encoding a SMN protein or variant thereof; and a second nucleic acid region comprising one or more target segment(s) of one or more endogenous microRNA(s), wherein the second nucleic acid region is at 3′ of the first nucleic acid region.

There is provided a method of identifying/characterising coronavirus infection in a human subject. In a specific embodiment the method comprising contacting a serum sample from the subject with an isolated host cell expressing a nucleotide that encodes for a codon optimised gene for SARS-CoV-2 spike protein (S protein); and detecting a binding of an antibody or an antigen-binding fragment thereof to said host cell using a flow cytometry and a labelled anti-human secondary antibody. Also disclosed is a method of identifying a subject having immunity for coronavirus, especially SARS-CoV-2 wherein a sample from a subject is incubated with said cell and is detected using the flow cytometry and the labelled anti-human secondary antibody. Nucleic acid encoding a SARS-CoV-2 S protein, viral vectors and host cells thereof are also disclosed.

The invention relates to vectors comprising two or more homologous nucleotide sequences and methods for generating them. The invention concerns substituting bases in the homologous nucleotide sequences with different bases that do not alter the encoded amino acid sequence. The invention allows for the reduction of intramolecular recombination between homologous nucleotide sequences, in particular in mammalian cells. The invention further relates to nucleotide sequences containing substituted bases.