Abstract: The present invention relates to a polynucleotide comprising a Complement Factor I (CFI) nucleotide sequence encoding a CFI polypeptide or a fragment thereof. The invention further relates to a viral particle comprising a recombinant genome comprising the polynucleotide of the invention, and a composition comprising the polynucleotide or viral particle of the invention. The invention also relates to methods of using, and uses of, the polynucleotide, viral particle and/or composition of the invention. The invention also relates to methods of using, and uses of, a polynucleotide comprising a CFI nucleotide sequence, a viral particle comprising a recombinant genome comprising the polynucleotide, or a composition comprising the polynucleotide or viral particle.

Abstract: The present invention relates to an improved assay and in particular to an improved assay that is capable of consistently measuring antibody titre, especially neutralising antibody (NAb) titre, at lower thresholds and/or with greater speed than conventionally-known assays. The invention further relates to use of such assays in combination with the provision of gene therapy and/or in combination with the provision of methods aimed at removal/depletion of neutralising antibodies from a patient.

Abstract: The present invention provides methods for inhibiting complement activation and uses thereof. More specifically, the present invention provides methods for inhibiting complement activation using inorganic polyphosphates of at least 10 phosphate units. The polyphosphates inhibit complement activation by one or more of: binding to the C6 complement protein, C1-esterase inhibitor (C1-inh), factor H or factor B; enhancing the activity of C1-inh; interfering with C1s-mediated cleavage of C2; destabilizing the C5b-6 complement protein complex; interfering with C5b,6 interaction with C7; interfering with binding of C5b-7 to a cell membrane; interfering with integration of C5b-7 into a cell membrane; interfering with binding of C5b-8 to a cell membrane; interfering with integration of C5b-8 into a cell membrane; destabilizing the membrane attack complex (MAC); or reducing the amount of C5b-9 deposited on a cell surface.

Abstract: The present invention provides methods for inhibiting complement activation and uses thereof. More specifically, the present invention provides methods for inhibiting complement activation using inorganic polyphosphates of at least 10 phosphate units. The polyphosphates inhibit complement activation by one or more of: binding to the C6 complement protein, C1-esterase inhibitor (C1-inh), factor H or factor B; enhancing the activity of C1-inh; interfering with C1s-mediated cleavage of C2; destabilizing the C5b-6 complement protein complex; interfering with C5b,6 interaction with C7; interfering with binding of C5b-7 to a cell membrane; interfering with integration of C5b-7 into a cell membrane; interfering with binding of C5b-8 to a cell membrane; interfering with integration of C5b-8 into a cell membrane; destabilizing the membrane attack complex (MAC); or reducing the amount of C5b-9 deposited on a cell surface.

Abstract: The present invention provides methods for inhibiting complement activation and uses thereof. More specifically, the present invention provides methods for inhibiting complement activation using polyphosphates.