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

Nucleic acid molecules such as shRNA dusters and artificial miRNA dusters are disclosed. Also disclosed are methods of use, compositions, cells, viral particles, and kits relating to the nucleic acid molecules disclosed herein.

The present invention provides compositions and methods for transducing cells (e.g. T cells or immune cells). Also provided herein are methods of treating a disease in a subject in need thereof.

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

Nucleic acid molecules such as shRNA clusters and artificial miRNA clusters are disclosed, Also disclosed are methods of use, compositions, cells, viral particles, and kits relating to the nucleic acid molecules disclosed herein. The disclosure provides, at least in part nucleic acid molecules such as shRNA clusters encoding shRNA-like molecules and artificial miRNA clusters encoding modified pri-miRNA-like molecules. The shRNA clusters and artificial miRNA clusters disclosed herein can be used, for example, to produce artificial RNA molecules, e.g., RNAi molecules. Cells, viral particles, compositions (e.g., pharmaceutical compositions), kits, and methods relating to the nucleic acid molecules, e.g., shRNA clusters and artificial miRNA clusters, are also disclosed. The nucleic acid molecules (e.g., shRNA clusters and artificial miRNA clusters), artificial RNA molecules (e.g., RNAi molecules), cells, viral particles, compositions (e.g., pharmaceutical compositions), and kits and methods disclosed herein can be used to treat or prevent a disease, e.g., HIV infection and/or AIDS.

The present disclosure relates to tandem expression cassettes encoding chimeric engulfment receptor molecules and chimeric antigen receptors/or T cell receptor binding proteins, host cells modified to include the tandem expression cassettes, and methods of making and using such receptor molecules and modified cells.

A process for producing blood coagulation Factor VII in large scale in 3 human cell lines (HepG2, Sk-Hep, HKB-11) and to select the best recombinant protein producer is described. The murine line BHK-21 was used as control. The data allowed for the assertion that the system used to modify cell lines was efficient, so that all the cells were satisfactorily modified, and produced the protein of interest in a stable form. In addition, when comparing the murine line BHK-21 with the human cells (HepG2, Sk-Hep-1 and HKB-11), the latter proved to be able to produce rFVII more efficiently, which allows us to conclude that human cell lines are a great alternative to produce recombinant blood coagulation factors in large scale.

The present invention relates to a chimeric antigen receptor comprising: a binding domain, the full DAP 10 protein, the full DAP 12 protein, or a functional variant thereof, anda hook binding domain.The present invention also relates to a vector system comprising one or more vector comprising: a nucleic acid comprising a nucleic acid sequence encoding a chimeric antigen receptor and optionally a nucleic acid encoding a hook fusion protein, preferably comprising a streptavidin core; wherein said nucleic acids are located on the same or on different vectors; The invention also encompasses lentiviral vector particles system, host cell and kit comprising said nucleic acids or vector system. The invention also relates to lentiviral vector particles system, host cell and kit as herein disclosed for their use as a medicament, notably for immunotherapy.

A lentiviral vector system for expressing a lentiviral particle is disclosed. The lentiviral vector system includes a therapeutic vector. The lentiviral vector system produces a lentiviral particle for upregulating PAH expression in the cells of a subject afflicted with phenylketonuria (PKU).

A method is provided to enhance repair or regeneration of a mammalian cardiovascular system to include heart and/or vasculature comprising: administering to a mammal in need thereof a composition comprising an effective amount of an agent that elevates levels of Smo, Ptc1, Shh, Ihh, Dhh, Gli1, Gli2, or Mycn,