Methods and products for transfecting cells

摘要

The present invention relates in part to nucleic acids encoding proteins, nucleic acids containing non-canonical nucleotides, therapeutics comprising nucleic acids, methods, kits, and devices for inducing cells to express proteins, methods, kits, and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods for inducing cells to express proteins and for reprogramming and gene-editing cells using RNA are disclosed. Methods for producing cells from patient samples, cells produced using these methods, and therapeutics comprising cells produced using these methods are also disclosed.

主权项

1. A method for treating HIV infection in a human subject, comprising:
(a) harvesting a hematopoietic cell from the subject; (b) transfecting the hematopoietic cell with an in vitro transcribed synthetic RNA molecule encoding a gene-editing protein for translation in a mammalian cell wherein:
(i) the hematopoietic cell is induced to express the gene-editing protein;(ii) the gene-editing protein causes a double-strand break in the DNA of the hematopoietic cell; and(iii) the double-strand break reduces the function of a gene selected from: CCR5 and CXCR4 to render the hematopoietic cell resistant to HIV infection; and (c) administering the HIV-resistant hematopoietic cell to the subject to result in the treatment of HIV infection in the subject.