Provided herein are compositions, systems, and methods for delivering cargo to a target cell. The compositions, systems, and methods comprise one or more polynucleotides encoding one or more endogenous retroviral elements for forming a delivery vesicle and one or more capture moieties for packaging a cargo within the delivery vesicle. The one or more endogenous retroviral elements for forming a delivery vesicle may comprise two or more of a retroviral gag protein, a retroviral envelope protein, a retroviral reverse transcriptase or a combination thereof. The retroviral gag protein alone, the retroviral envelope protein alone, or both the retroviral gag protein and retroviral envelope protein may be endogenous.

Provided is a nucleic acid construct for expressing a gene of interest, the nucleic acid construct comprising, in order from 5′-terminus, each sequence of: (a) a 5′ long terminal repeat (LTR) sequence derived from a retrovirus; (b) a packaging signal sequence (IV) derived from a retrovirus; (c) a sequence or multiple cloning site of the gene of interest; (d) a post-transcriptional regulatory sequence (PRE); (e) an siRNA generating sequence which forms at least one stem-loop structure and in which RNA, which induces RNA interference in mammalian cells, is transcribed; and (f) a 3′ LTR sequence derived form a retrovirus.

There are provided compositions and methods for modulating stem cell division, in particular, division symmetry. It has been demonstrated that Wnt7a polypeptide fragments promoting symmetrical expansion of stem cells. The compositions and methods of the invention are useful, for example, in modulating stem cell division symmetry in vitro, ex vivo, and in vivo, in replenishing and expanding the stem cell pool, and in promoting the formation, maintenance, repair and regeneration of tissue.

The present invention relates to a virus-derived particle comprising one or more Cas protein(s), as well as to kits and methods using the same for altering a target nucleic acid.

Human-derived virus-like particles (heVLPs), comprising a membrane comprising a phospholipid bilayer with one or more HERV-derived envelope proteins on the external side; one or more HERV-derived GAG proteins in the heVLP core, and a cargo molecule, e.g., a biomolecule and/or chemical cargo molecule, disposed in the core of the heVLP on the inside of the membrane, wherein the heVLP does not comprise a gag protein, except for gag proteins that are encoded in the human genome or gag proteins that are encoded by a consensus sequence that is derived from gag proteins found in the human genome, and methods of use thereof for delivery of the cargo molecule to cells.

The present invention aims to provide a method for preparing urothelial cells that can be applied to the treatments of urologic diseases, particularly, diseases caused by urothelial cell damage, diseases caused by loss of urothelial cells and dysfunction, and the like, urothelial cells prepared by said method, and a medium for inducing (generating) urothelial cells. Urothelial cells prepared by a method for inducing a urothelial cell, including a step of introducing at least one member selected from the group consisting of

FOXA1 (Forkhead box A1) gene or an expression product thereof,
TP63 (tumor protein P63) gene or an expression product thereof,
MYCL (L-Myc) gene or an expression product thereof, and
KLF4 (Kruppel-like factor 4) gene or an expression product thereof
to a mammalian somatic cell as an exogeneous factor can be applied to the treatment of urologic diseases.

The present application relates to the field of immunotherapy, more particularly to the field of chimeric antigen receptors (CARs). Here, CARs are proposed that are directed against B-cell Maturation Antigen (BCMA, also known as CD269). Also proposed are polynucleotides, vectors encoding the transmembrane polypeptide chains and cells expressing such CARs. These cells are particularly suitable for use in immunotherapy, and strategies to treat diseases such as cancer using these cells are also provided. The engineered immune cells, such as T-cells or natural killer (NK) cells, expressing such CARs are particularly suitable for treating lymphomas, multiple myeloma and leukemia.

The present invention relates to a virus-derived particle comprising one or more Cas protein(s), as well as to kits and methods using the same for altering a target nucleic acid.

This disclosure provides a retroviral replicating vector for gene delivery comprising a therapeutic cassette containing at least one mini-promoter linked to a gene to be expressed.

The present invention relates to a virus-derived particle comprising one or more Cas protein(s), as well as to kits and methods using the same for altering a target nucleic acid.