Abstract: The present invention relates to polynucleotides comprising a Factor IX nucleotide sequence, wherein the Factor IX nucleotide sequence comprises a coding sequence that encodes a Factor IX protein or fragment thereof and wherein a portion of the coding sequence is not wild type. The present invention further relates to viral particles comprising a recombinant genome comprising the polynucleotide of the invention, compositions comprising the polynucleotides or viral particles, and methods and uses of the polynucleotides, viral particles or compositions.

Abstract: A sensor device is provided to sample data from a fluid in a sealed environment. The sensor comprises a housing and a diamond within the housing. The housing is formed such that the device is reversibly insertable into the sealed environment so that the diamond directly interfaces with the sealed environment.

Abstract: A method of diagnosing Frontotemporal dementia (FTD) in a subject in need thereof is provided. The method comprising detecting a level of at least one micro RNA (miR) selected from the group consisting of hsa-miR-361-5p, hsa-miR-629-5p, hsa-miR-628-3p, hsa-miR-379-5p, hsa-miR-1-3p, hsa-miR-26a-5p, hsa-miR-125a-5p, hsa-miR-125b-5p, hsa-miR-142-5p and hsa-miR-340-5p in a biological sample of the subject, wherein when said level of said at least one micro RNA (miR) is higher than that in a control sample, it is indicative of FTD.

Abstract: The present invention provides a bi-specific molecule which comprises: (i) a first domain which binds B cell maturation antigen (BCMA) and comprises at least part of a proliferation-inducing ligand (APRIL); and (ii) a second domain capable of activating a T cell. The invention also provides the use of such a molecule in the treatment of plasma-cell mediated diseases, such as multiple myeloma.

Abstract: A membrane-spanning nanopore is provided that comprises: i. at least one scaffold polynucleotide strand; ii. a plurality of staple polynucleotide strands; and iii. at least one hydrophobically-modified polynucleotide strand, wherein the at least one hydrophobically-modified polynucleotide strand comprises a polynucleotide strand and a hydrophobic moiety; wherein each of the plurality of staple polynucleotide strands hybridises to the at least one scaffold polynucleotide strand to form the three-dimensional structure of the membrane-spanning nanopore, and wherein the at least one hydrophobically-modified polynucleotide strand hybridises to a portion of the at least one scaffold polynucleotide strand, the membrane-spanning nanopore defining a central channel with a minimum internal width of at least about 5 nm. Membranes comprising the membrane-spanning nanopore and applications of those membranes are also provided.

Abstract: There is described Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) antibodies that specifically bind a ROR1 polypeptide, and their use. In particular, isolated monoclonal antibodies are described and their use in a number of applications, including in the detection, prevention and treatment of cancer.

Abstract: The present invention relates to the field of medicine. In particular, it relates to novel antisense oligonucleotides that may be used in the treatment, prevention and/or delay of Stargardt disease.

Abstract: There is provided a nucleic acid molecule comprising a nucleotide sequence encoding a Factor VIII protein, wherein a B domain portion of the Factor VIII protein is encoded by a nucleotide sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a Factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating Haemophilia for example, Haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.

Abstract: A compound having the formula (I) R1-COOH. R1 is an alkyl or alkenyl group having a C7-11 backbone, optionally branched with a C1-6 alkyl group at any C position in the backbone, or a pharmaceutically acceptable salt, amide or ester thereof. The backbone of the alkyl or alkenyl group, and/or the branched alkyl groups, are optionally interrupted by one or more heteroatoms, provided that when R1 is an alkyl group having a C7 backbone, the branching does not consist only of a hexyl group at the a carbon of R1, or only of a methyl group at the ? carbon of R1, or of only single methyl groups at both the ? and ?-1 carbons of R1, and provided that when R1 is an alkyl group having a C8 or C11 backbone, the branching does not consist only of a propyl group at the a carbon of R1.

Abstract: There is described a multiphasic osteochondral scaffold for osteochondral defect repair, the scaffold comprising a bone phase and a cartilage phase, wherein the bone phase comprises a support matrix and the cartilage phase comprises a polymeric matrix, and the scaffold comprises a non-porous layer between the bone phase and the cartilage phase. Also described is a multiphasic osteochondral scaffold for osteochondral defect repair, the scaffold comprising a bone phase and a cartilage phase, wherein the bone phase comprises a support matrix and the cartilage phase comprises a polymeric matrix, and wherein the support matrix is tapered so that the dimensions of the support matrix are less at the lower end of the support matrix than at the upper end of the support matrix.

Abstract: The present disclosure provides methods of generating multiplexed genetically modified animals, for example, porcine endogenous retrovirus (PERV)-inactivated pigs. The disclosure also provides methods of improving the birth rate of multiplexed genetically modified animals. In some embodiments, the present closure is concerned with the generation and utilization of porcine cells in which porcine endogenous retroviral (PERV) elements have been inactivated. In sonic embodiments, the PERV-free or PERV-reduced porcine cells are cloned to produce porcine embryos. In some embodiments, the PERV-free or PERV-reduced embryos may be grown into adult swine from which organs and/or tissues may be extracted and used for such purposes as xenotransplantation into non-porcine animals such as humans.

Abstract: A method of prognosing a course of disease progression and/or survival time in a subject diagnosed with ALS or FTD is disclosed. The method comprising: (a) detecting a level of miR-181 in a biological sample of the subject; and (b) determining the disease progression and/or survival time based on the level of the miR-181, wherein: (i) when the level of miR-181 is higher than that in a control sample, it is indicative of a rapid disease progression and/or poor survival; or (ii) when the level of miR-181 is about the same or lower than that in the control sample, it is indicative of a slow disease progression and/or good survival. Methods of prognosing a stage of disease in a subject diagnosed with ALS or FTD are also disclosed.

Abstract: Herein is disclosed, a rechargeable flow battery, wherein the flow battery comprises: first and second electrodes, separated such that ions are allowed to flow between them, wherein a first reservoir comprising or for holding a first fluid electrolyte is fluidly connected to the first electrode, to allow circulation of the first fluid electrolyte from the first reservoir to the first electrode and from the first electrode to the first reservoir; and a first current collector comprising a layer of electrically conductive material having opposing first and second sides, wherein the first electrode is disposed on the first side of the first current collector, such that electrons can flow from the electrode to the first current collector, and a first layer of dielectric material is disposed on the second side of the first current collector.

Abstract: A composition comprising a decanoic acid to octanoic acid ratio of 70:30 to 90:10 wt/wt for use in treating epilepsy or controlling epileptic seizures.

Abstract: The invention relates to a new, more potent, coagulation Factor IX (FIX) expression cassette for gene therapy of Haemophilia B (HB). Disclosed is a vector for expressing factor IX protein, the vector comprising a promoter, a nucleotide sequence encoding for a functional factor IX protein, and an intron sequence, wherein the intron sequence is positioned between exon 1 and exon 2 of the nucleotide sequence encoding for a functional factor IX protein, and wherein the intron sequence has at least 80% identity to the sequence of SEQ ID NO. 1 as disclosed herein.

Abstract: The present invention is directed to a method for producing nanoparticles and microparticles composed of peptide- or peptoid-containing amphiphilic polymers. The method is simple, capable of achieving high yields, and can be tailored to produce a range of industrially and therapeutically useful structures including vesicles, micelles and hydrogels. The present invention also provides related hydrogels and vesicles having beneficial properties such an ability to degrade and release a payload in response to external stimuli.

Abstract: There is described bispecific antibodies which selectively bind to Receptor Tyrosine Kinase Like Orphan Receptor 1 (ROR1) and the CD3 subunit of the T-Cell Receptor (TCR), their production and their use. Also described is the use of the bispecific antibodies in the treatment of cancer.

Abstract: The present invention is directed to a nanoparticle or microparticle for binding to the surface of a cell, wherein the nanoparticle or microparticle comprises (i) multiple different ligand types on its external surface which are capable of binding to different respective receptor types on said cell surface, and (ii) a polymer brush on its external surface. The present invention is further directed to pharmaceutical compositions comprising a plurality of nanoparticles or microparticles of the invention, medical uses of such nanoparticles or microparticles, and a vaccine comprising such nanoparticles or microparticles.

Abstract: Disclosed herein are methods of treating and/or preventing portal hypertension and/or restoring liver function using L-ornithine phenylacetate.

Abstract: A computer system and corresponding method are used to assist in planning a trajectory for a surgical insertion into a skull to a target representing an anatomical region.