Fusogenic lipid nanoparticles and methods for the manufacture and use thereof for the target cell-specific production of a therapeutic protein and for the treatment of a disease, condition, or disorder associated with a target cell

A system for the targeted production of a therapeutic protein within a target cell, said system comprising:

• a. a vector that is capable of delivering a nucleic acid to a cell, said vector comprising an expression construct;
• and
• b. an expression construct for the targeted production of a therapeutic protein within a target cell, said expression construct comprising:
• i. a transcriptional promoter that is activated in response to one or more factors each of which is produced within said target cell;
• and
• ii. a nucleic acid that is operably linked to and under regulatory control of said transcriptional promoter, wherein said nucleic acid encodes a therapeutic protein that can reduce, prevent, and/or eliminate the growth and/or survival of a cell, including said target cell.

A method for reducing, preventing, and/or eliminating the growth of a target cell, said method comprising contacting a target cell with a system for the targeted production of a therapeutic protein within said target cell, said system comprising:

• a. a vector that is capable of delivering a nucleic acid to a cell, said vector comprising an expression construct;
• and
• b. an expression construct for the targeted production of a therapeutic protein within a target cell, said expression construct comprising (i) a transcriptional promoter that is activated in response to one or more factors each of which is produced within said target cell;
• and (ii) a nucleic acid that is operably linked to and under regulatory control of said transcriptional promoter, wherein said nucleic acid encodes a therapeutic protein,
• wherein production of said therapeutic protein in said target cell reduces, prevents, and/or eliminates the growth and/or survival of said target cell.

A method for the treatment of a disease or condition in a patient having a target cell, said method comprising:

• administering to said patient a system for the targeted production of a therapeutic protein within a target cell, said system comprising
• a. a vector that is capable of delivering a nucleic acid to a cell, said vector comprising an expression construct;
• and
• b. an expression construct for the targeted production of a therapeutic protein within a target cell, said expression construct comprising (i) a transcriptional promoter that is activated in response to one or more factors each of which is produced within said target cell;
• and (ii) a nucleic acid that is operably linked to and under regulatory control of said transcriptional promoter, wherein said nucleic acid encodes a therapeutic protein, wherein production of said therapeutic protein in said target cell reduces, prevents, and/or eliminates the growth and/or survival of said target cell thereby slowing, reversing, and/or eliminating said disease or condition in said patient.