A method of producing renal progenitor cells from pluripotent stem cells involves culturing pluripotent stem cells in a medium containing FGF2, BMP4, a GSK-3β inhibitor, and retinoic acid or a derivative thereof, culturing the resulting cells in a medium containing FGF2, a GSK-3β inhibitor, and BMP7, culturing the resulting cells in a medium containing FGF2, a GSK-3β inhibitor, BMP7, and a TGFβ inhibitor, culturing the resulting cells in a medium containing FGF2, a GSK-3β inhibitor, BMP7, activin, and a ROCK (Rho-kinase) inhibitor, culturing the resulting cells in a medium containing retinoic acid or a derivative thereof, and FGF9, and culturing the resulting cells in a medium containing a GSK-3β inhibitor and FGF9, to induce renal progenitor cells from intermediate mesodermal cells.

The present invention relates to in situ glycosylated MHC II/CII peptide complexes, i.e., complexes naturally glycosylated during recombinant protein expression in the host cell. The invention further relates to methods of producing glycosylated MHC II/CII peptide complexes in mammalian cells. Furthermore the invention relates to the use of such post-translationally modified, preferably glycosylated MHC II/CII complexed for use in treating rheumatoid arthritis, preferably in humans.

There are disclosed functional nucleic acid molecules comprising at least one target binding sequence comprising a sequence reverse complementary to a frataxin mRNA sequence; and a regulatory sequence comprising a SINE B2 element or a functionally active fragment of a SINE B2 element or an internal ribosome entry site (IRES) sequence or an IRES derived sequence.

The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.

Provided is a proteolysis-targeting virus, wherein one or more proteolysis-targeting molecules that can be recognized by the ubiquitin-proteasome system are comprised at one or more different sites of protein thereof, and the viral protein is linked to the proteolysis-targeting molecules by one or more linkers that can be selectively cleaved. Also provided are a nucleic acid molecule encoding the proteolysis-targeting virus, a nucleic acid vector expressing the proteolysis-targeting virus, a preparation method for the proteolysis-targeting virus, methods for the preparation of an attenuated live virus, replication-incompetentlive virus, replication-controllable live virus, and a relevant vaccine and medication for preventing and treating virus infections, a vaccine or pharmaceutical composition comprising the proteolysis-targeting virus, and a system for preparing the proteolysis-targeting virus.

The disclosure describes novel systems, methods, and compositions for the manipulation of nucleic acids in a targeted fashion. The disclosure describes non-naturally occurring, engineered CRISPR systems, components, and methods for targeted modification of nucleic acids. Each system includes one or more protein components and one or more nucleic acid components that together target nucleic acids.

The present invention relates to high throughput in vitro genetic manipulation. In particular, it relates to scalable CRISPR gene insertions in mammalian cells.

This invention relates to retroviral gene transfer vectors, particularly lentiviral vectors, pseudotyped with hemagglutinin-neuraminidase (HN) and fusion (F) proteins from a respiratory paramyxovirus, comprising a promoter and a transgene; and methods of making the same. The present invention also relates to the use of said vectors in gene therapy, particularly for the treatment of respiratory tract diseases such as Cystic Fibrosis (CF).

Region-specific extracellular matrix (ECM) biomaterials are provided. Such materials include acellular scaffolds, sponges, solutions, and hydrogels suitable for stem cell culture.

The present invention provides a virus-like particle (VLP) having a viral envelope which comprises: (i) a membrane protein comprising the extracellular domain of CD86; and (ii) a CD3-binding membrane protein. The VLP may be used to activate T cells prior to viral transduction.