COMPOSITIONS AND METHODS FOR EFFICIENT AND STABLE GENETIC MODIFICATION OF EUKARYOTIC CELLS

Abstract: compositions and methods for efficient cellular genomic engineering that transduce diverse cell types with minimal toxicity, leading to efficient and stable genomic modifications are described. the compositions and methods are applicable to development of chimeric antigen receptor engineered t cell therapy (car-t). an exemplary method introduces a gene of interest into cells by introducing to the cell a viral vector including a transposon encoding the gene of interest and mrna including a sequence that encodes transposase enzymes configured to mediate targeted integration of the transposon into the cellular genome, whereby the mrna is introduced to the cell via electroporation. also disclosed are genetically modified cells and pharmaceutical compositions and methods of use thereof for treating subjects having diseases or disorders.

Inventor(s): Sidi Chen, Lupeng Ye, Stanley Lam

CPC Classification: A61K40/11 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

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