Provided is a chimeric antigen receptor targeting CLL1 and an application thereof. The chimeric antigen receptor targeting CLL1 comprises an antigen binding domain, a hinge region, a transmembrane domain and a signal transduction domain; the antigen binding domain is an anti-CLL1 antibody. The present application uses an anti-CLL1 antibody as the antigen binding domain to construct a chimeric antigen receptor molecule, the chimeric antigen receptor targeting CLL1 has specific targeting effect on CLL1 positive tumor cells, and immune cells expressing chimeric antigen receptor targeting CLL1 have a significant killing effect in vitro and in vivo, and secrete a large amount of cytokine IFN-γ after co-cultured with CLL1 positive tumor cells, which has a specific clearance effect on CLL1 positive tumor cells.

Provided is a viral particle comprising a genomic RNA suitable for delivering a polynucleotide sequence of interest (SOI) into a cell and/or a subject. The genomic RNA comprises, from 5′ to 3′: (a) the SOI replacing the upstream R, (b) a U5, (c) a primer binding site (PBS), (d) an encapsidation signal (Psi), (e) polypurine tract(s) (PPT), and (f) a U3. The SOI preferably takes the form of single-stranded DNA or a DNA-RNA hybrid after initiation of reverse transcription. Additionally provided are polynucleotides, vectors, cells, components, compositions, kits, methods and uses of the viral particle.

Recombinant vectors containing at least: a backbone comprising essential sequences for integration into a target cell genome; a nucleic acid encoding a CCR5 RNAi operatively linked to a a first expression control element that regulates expression of the nucleic acid encoding the RNAi of the CCR5; a nucleic acid encoding at least the extracellular domain of CD25 operatively linked to a second expression control element that regulates expression of the nucleic acid encoding at least the extracellular domain of CD25 are provided by this disclosure. In an alternative aspect, the vector also contains polynucleotides encoding TRIM5alpha and HIV TAR decoy sequences along with gene expression regulation elements such as promoters operatively linked to the polynucleotides. The vectors are combined with packaging plasmid and envelope plasmids and optionally conjugated to cell-specific targeting antibodies. Diagnostic and therapeutic methods for using the compositions are further provided herein.

The invention provides Natural Killer (NK) cells that have a reduced or ablated Signal Regulatory Protein Alpha (SIRPα-) function when compared to a NK cell having an unmodified SIRPα-function that effectively kills a population of cancer cells that express CD47.

Disclosed herein are compositions of retroviruses and methods of using the same for gene delivery to a hematopoietic stem cell (HSC), wherein the retroviruses comprise a viral envelope protein comprising at least one mutation that diminishes its native function, a non-viral membrane-bound protein comprising a membrane-bound domain and an extracellular targeting domain.

The present specification provides a method of producing induced functional astrocytes (iAs) from human pluripotent stem cells substantially more rapidly than previously achieved. These iAs express biomarkers and have functional characteristics typical of natural astrocytes. The iAs are useful in the exploration of astrocyte biology, pathophysiology, and in models of neurologic diseases and disorders.

Described herein are immune cells comprising: a chimeric antigen receptor (CAR) that comprises (i) an extracellular target-binding domain comprising an antibody moiety; (ii) a transmembrane domain; and (iii) a primary signaling domain, and a chimeric stimulating receptor (CSR) that comprises (i) a ligand-binding module that is capable of binding or interacting with a target ligand; (ii) a transmembrane domain; and (iii) a CD30 costimulatory domain, in which the CSR in the immune cells lacks a functional primary signaling domain. Also provided herein are methods of using the same or compositions thereof for therapeutic treatment of cancers (e.g., hematological cancers or solid tumor cancers).

Provided herein are methods for enhancing the transduction efficiency of vectors into cells, e.g., primary human T lymphocytes.

Objects to be achieved are to provide a nerve cell with which it is possible to visualize and quantify the intracellular tau without using the exogenous promoter and to provide a pluripotent stem cell with which the nerve cell can be produced, to provide a method of screening a substance, including using the pluripotent stem cell or nerve cell described above, and a substance screened by the above method, and to provide a kit including a targeting vector and a gRNA.

There is provided a pluripotent stem cell including a DNA encoding a reporter molecule, the DNA being introduced adjacent to an endogenous tau gene such that a tau protein is expressed as a fusion protein fused with a reporter molecule.

The disclosure provides tandem arrays of CRISPR RNAs and methods of use.