The present invention provides a genetically modified NK cell line prepared by transducing host NK cell line with a gene construction encoding a cancer antigen-specific chimeric antigen receptor (CAR) comprising a FLT3-specific monoclonal antibody or a functional fragment thereof, a transmembrane domain, and a CD3ζ domain of a T-cell receptor for more efficient immunotherapy of acute myeloid leukemia, and use thereof, for more efficient immunotherapy of acute myeloid leukemia.

Described herein are methods for transfecting mesenchymal stem cells (MSCs) with a nucleic acid construct using a cationic polymer, a first reagent capable of redirecting endocytosed nucleic acids from intracellular acidic compartments, and a second agent capable of stabilizing a microtubular network of the MSCs. The methods are free of virus-based transfection vehicle materials and the transfected MSCs have substantially unchanged multipotent phenotype. In certain embodiments, the transfected MSCs express functional genes comprising suicide gene, such as cytosine deaminase or uracil phosphoribosyltransferase. Also described are methods for the treatment of diseases, such as cancer, using such transfected cells in combination with 5FC, 5FU, GCV, as well as kits and composition relating thereof.

A cell preparation for treating brain tumors used in combination with a prodrug that is converted to 5-fluorouracil by cytosine deaminase, wherein the cell preparation comprises neural stem cells derived from pluripotent stem cells having a cytosine deaminase gene and a uracil phosphoribosyltransferase gene is provided to establish new means for treating brain tumors.

The present invention provides an oncolytic virus comprising nucleotide sequence(s) encoding one or more immune checkpoint modulator(s). It also concerns a pharmaceutical composition comprising effective amount of said oncolytic virus and, eventually, a pharmaceutically acceptable vehicle and its use for treating proliferative diseases such as cancers.

The present invention provides an oncolytic virus comprising nucleotide sequence(s) encoding one or more immune checkpoint modulator(s). It also concerns a pharmaceutical composition comprising effective amount of said oncolytic virus and, eventually, a pharmaceutically acceptable vehicle and its use for treating proliferative diseases such as cancers.

The present invention provides an oncolytic virus comprising nucleotide sequence(s) encoding one or more immune checkpoint modulator(s). It also concerns a pharmaceutical composition comprising effective amount of said oncolytic virus and, eventually, a pharmaceutically acceptable vehicle and its use for treating proliferative diseases such as cancers.

The present invention provides new and improved methods for performing in situ transcriptomics from rare cells of interest present in complex multicellular environments. These methods involve expressing a recombinant cytosine deaminase enzyme in the cells of interest, and also supplying an exogenous non-naturally occurring halogenated cytosine substrate for the enzymewhich results in the generation halogenated uridine which is incorporated into RNA, thereby tagging RNA in the cells of interest. The invention also provides several variations of such methods that significantly improve the sensitivity and specificity of the RNA labeling. Furthermore, the invention also provides simple and efficient methods for purifying the tagged RNA. The purified tagged RNA can be used to analyze the transcriptomes of the cells of interest by RNA-sequencing and other methods.

To treat brain dysfunctions, brain diseases or tumors, the present invention provides a cell preparation comprising neural stem cells differentiated from pluripotent stem cells into which a suicide gene has been introduced.

A cell preparation for treating brain tumors used in combination with a prodrug that is converted to 5-fluorouracil by cytosine deaminase, wherein the cell preparation comprises neural stem cells derived from pluripotent stem cells having a cytosine deaminase gene and a uracil phosphoribosyltransferase gene is provided to establish new means for treating brain tumors.

Methods are provided for differential biosynthetic labeling of RNA, including identification of cell type-specific programs of gene expression. The methods and compositions of the invention allow detection and/or purification of RNAs with precise spatial and temporal resolution. In various embodiments of the invention, the methods are applied to animal cells, including cell lines, stem cells, selected lineages of organisms, and the like.