Abstract: The present invention relates to gene therapy agents for the treatment of pulmonary alveolar proteinosis (PAP), particularly autoimmune PAP (aPAP). In particular, the present invention relates to gene therapy vectors which drive transient and/or low-level expression of granulocyte-macrophage colony-stimulating factor (GM-CSF), which provide a therapeutic effect without therapy-associated toxicity. The invention further relates to related products and an animal model of aPAP.

Abstract: The present invention relates to methods for the production of immune cells and immunomodulatory cells expressing a transgene of interest, using lentiviral vectors. The invention also relates to populations of cells produced by such methods and uses thereof.

Abstract: The present invention relates to nucleic acid cassettes for gene therapy, particularly to nucleic acid cassettes encoding therapeutic proteins in combination with exogenous signal peptides. The invention further relates to viral and non-viral vectors comprising such nucleic acid cassettes, and the use of such nucleic acid cassettes and vectors to increase expression of therapeutic proteins by airway cells.

Abstract: The present invention relates to combination treatments for cystic fibrosis, particularly combinations of modulators of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and gene therapy.

Abstract: A nucleic acid delivery complex is provided which comprises a condensed polypeptide/nucleic acid complex and a cationic lipid wherein the complex comprises (a) a nucleic acid sequence of interest (NOI); and (b) one or more viral nucleic acid packaging polypeptides, or derivatives thereof, said polypeptides or derivatives thereof being (i) capable of binding to the NOI; and (ii) capable of condensing the NOI; and wherein the NOI is heterologous to the polypeptide. Also provided is a method of introducing an NOI into a cell using the delivery vector.

Abstract: A nucleic acid delivery complex is provided which comprises a condensed polypeptide/nucleic acid complex and a cationic lipid wherein the complex comprises (a) a nucleic acide sequence of interest (NOI); and (b) one or more viral nucleic acid packaging polypeptides, or derivatives thereof, said polypeptides or derivatives thereof being (i) capable of binding to the NOI; and (ii) capable of condensing the NOI; and wherein the NOI is heterologous to the polypeptide. Also provided is a method of introducing an NOI into a cell using the delivery vector.