Provided is a genetically engineered immune effector cell, wherein the immune effector cell expresses a receptor specifically recognizing a target antigen and IL7, and the IL-7 is inducibly expressed and regulated by the receptor. Further provided are a nucleic acid molecule expressing the receptor and IL7 in the immune effector cell and a method for preparing the immune effector cell.

Methods for preparing highly purified rLV vector formulations at the scale needed to meet anticipated demand for human gene therapy are provided.

A method is provided for producing oligodendrocyte-like cells, including (A) increasing abundances of oligodendrocyte transcription factor 2 (OLIG2) mutant and SRY-box transcription factor 10 (SOX10) in human pluripotent stein cells and (B) culturing the human pluripotent stem cells in which the abundances of the OLIG2 mutant and the SOX10 are increased and consequently differentiating the human pluripotent stem cells into oligodendrocyte-like cells, in which the OLIG2 mutant lacks a serine residue of wild-type OLIG2 at position 147, or the serine residue of the wild-type OLIG2 at position 147 is substituted with an amino acid other than serine.

The invention provides in vivo methods for identifying cancer-associated immunotherapy targets.

Provided are cells, such as engineered cells, that express a prostate-specific membrane antigen (PSMA) or a modified form thereof. In some embodiments, the cell further contains a genetically engineered recombinant receptor, such as a chimeric antigen receptor, that specifically binds to an antigen. The present disclosure also provides methods of detecting, identifying, selecting or targeting cells expressing PSMA, such as in connection with administration of such cells to subjects, including methods of adoptive cell therapy, or in connection with methods of manufacturing engineered cells.

The present invention relates to a method of treating, preventing or delaying the progression of cancer and/or tumour in a subject comprising administering to the subject a treatment regimen comprising an effective amount of modified immunoresponsive cells expressing or presenting a heterologous T-cell receptor (TCR) having the property of binding to MAGE A4

The present invention relates to a method of treating, preventing or delaying the progression of cancer and/or tumour in a subject comprising administering to the subject a treatment regimen comprising an effective amount of modified immunoresponsive cells expressing or presenting a heterologous T-cell receptor (TCR) having the property of binding to MAGE A4

A method of treating an inherited retinal disease (IRD) associated with a pathogenic point mutation in a mutant allele of an IRD-related gene in the retina or the retinal pigment epithelium (RPE) of a subject in need thereof includes base editing the pathogenic point mutation in the retinal cell or retinal pigment epithelium cell to correct the pathogenic mutation, generate a non-pathogenic point mutation, or modulate expression of an IRD-related gene and restore visual function of subject.

A system and method for gene editing by using an engineered cell are provided. The system includes the engineered cell embedded with a synthetic protein receptor and a target cell. The engineered cell contains a CRISPR/CasRx system and a sgRNA gene sequence. The synthetic protein receptor includes an extracellular target cell recognition domain, a native Notch core domain, an intramembranous hydrolyzable polypeptide and effectors. The extracellular target cell recognition domain can recognize antigen molecules on the target cell surface; and the effectors act as transcription factors for CasRx enzyme and sgRNAs. CasRx and gRNA are expressed in the engineered cell for gene editing to edit mRNA of the target cell. In this way, the application range of the engineered cell is expanded, the pertinence and specificity of gene editing are improved, the off-target effect is reduced, the collective non-specific reaction is reduced, and the safety of gene editing is improved.

Provided herein are polypeptides comprising membrane-anchored IL-12. Also provided herein are T cells expressing the membrane-anchored IL-12. Further, methods of treating cancer comprising administering T cells expressing membrane-anchored IL-12 are provided herein. Also provided are combination treatments comprising T cells expressing membrane-anchored IL-12 and T cell chemoattractant-inducing chemokines. In addition, methods are provided for activating T cells to express NKG2D and methods of their use in the treatment of cancer.