Methods and products for transfecting cells

Patent metadata

Field	Value
Publication number	US-2016333378-A1
Application number	US-201615222453-A
Country	US
Kind code	A1
Filing date	Jul 28, 2016
Priority date	Dec 5, 2011
Publication date	Nov 17, 2016
Grant date	—

Abstract

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.

First claim

1 .- 61 . (canceled)

62 . A therapeutic composition, comprising a gene-edited hematopoietic cell, wherein:
the gene-edited hematopoietic cell comprises:
an in vitro transcribed synthetic RNA molecule encoding a gene-editing protein that targets a gene selected from CCR5 and CXCR4;
a gene-editing protein-created double-strand break in its DNA, the double-strand break reducing the function of one or more of CCR5 and CXCR4; and
the therapeutic composition is suitable for introduction into a human subject with HIV/AIDS.

63 . The therapeutic composition of claim 62 , wherein the gene-editing protein comprises a DNA-binding domain and a catalytic domain of a nuclease.

64 . The therapeutic composition of claim 62 , wherein the gene-editing protein is selected from a TALEN and a zinc finger nuclease.

65 . The therapeutic composition of claim 62 , wherein the in vitro transcribed synthetic RNA molecule further comprises one or more of a 5′-cap, a 5′-cap 1 structure, and a 3′-poly(A) tail.

66 . The therapeutic composition of claim 62 , wherein the double-strand break is within about 5,000,000 bases of the transcription start site of the CCR5 or CXCR4 gene.

67 . The therapeutic composition of claim 62 , wherein the composition is suitable for conferring resistance to HIV infection in the subject.

68 . The therapeutic composition of claim 62 , wherein the human subject is infected with HIV.

69 . The therapeutic composition of claim 62 , wherein the human subject is afflicted with AIDS.

70 . The therapeutic composition of claim 62 , wherein the hematopoietic cell is a hematopoietic stem cell.

71 . The therapeutic composition of claim 62 , wherein the hematopoietic cell is a white blood cell.

Assignees

• Factor Bioscience Inc

Inventors

• Angel Matthew
• Rohde Christopher

Classifications

• A61P7/06

  Antianaemics · CPC title

• A61P9/10

  for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis · CPC title

• A61P43/00

  Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title

• A61P3/10

  for hyperglycaemia, e.g. antidiabetics · CPC title

• A61P31/18

  for HIV · CPC title