The present invention provides a process for recovering a viral product from a composition comprising said product and product-related impurities, which process comprises contacting the composition with a functionalised chromatography medium comprising one or more polymer nanofibres, wherein the viral product comprises a plurality of viruses, virus particles/virions, viral cores, membrane-stripped viruses, viral cores with outer membrane(s) removed and/or capsids removed, or proviruses, each of which contains one or more polynucleotides, and wherein the product-related impurities comprise a plurality of viruses, virus particles/virions, virus-like particles, viral cores, membrane-stripped viruses, viral cores with outer membrane(s) removed and/or capsids removed or proviruses, each of which is substantially devoid of polynucleotides.

The present invention relates to vectors containing liver-specific regulatory sequences and codon-optimized factor IX or factor VIII genes, methods employing these vectors and uses of these vectors. Expression cassettes and vectors containing these liver-specific regulatory elements and codon-optimized factor IX or factor VIII genes are also disclosed. The present invention is particularly useful for applications using gene therapy, in particular for the treatment of hemophilia A and B.

Drug compositions of angiogenesis therapy contain gene coding for human prostacyclin synthase (hPGIS) synthesizing prostaglandin I.sub.2 with activities of vasodialation and/or anti-platelet aggregation; drug compositions contain adeno-associated virus (AAV) inserted with gene for angiogenesis factors. The administration of the drug compositions into the aimed treatment region results in transfer of AAV type 1-hPGIS to skeletal muscles and induces a notable expression of human PGIS gene in skeletal muscles. The PGI.sub.2 is produced by mediation of the gene expression in the muscle cells, secreted, induces vessel-protective, neovascularization and anti-platelet aggregation actions, which lead to an improvement in vascular ischemia.

Nucleases and methods of using these nucleases for expressing a transgene from a safe harbor locus in a secretory tissue, and clones and animals derived therefrom.

The invention provides methods to inhibit or treat brain cancers by locally inhibiting expression or activity of growth factors or growth factor receptors.

Chimeric fusion proteins and polynucleotides encoding the chimeric fusion proteins are provided for the treatment of proliferative disorders, automimmune diseases and alloimmune responses. The chimeric fusion proteins comprise a CD24 extracellular domain, an EBV-induced 3 (EBI3) polypeptide subunit, and a p28 IL-27 polypeptide subunit, wherein the EBB polypeptide and the p28 IL-27 polypeptide subunit are covalently joined by a flexible peptide linker.

Methods and compositions are provided for editing the genome of a cell without the use of an exogenously supplied nuclease. Aspects of the methods include contacting a cell with a targeting vector comprising nucleic acid sequence to be integrated into the target locus, where the cell is not also contacted with a nuclease. In addition, reagents, devices and kits thereof that find use in practicing the subject methods are provided.

The invention relates to methods of treating melanoma using a herpes simplex virus in combination with an immune checkpoint inhibitor.

Described are nucleic acid regulatory elements that are able to enhance liver-specific expression of genes, methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These are particularly useful for applications using gene therapy.

The present invention relates, in general, to Pompe disease and, in particular, to a methods of treating Pompe disease and to compounds/constructs suitable for use in such methods.