IP Library Granted Patent US 10,000,757
Granted Patent B2
US 10,000,757 · App. 11/921,140 · Granted Jun 19, 2018

Gene vector

Inventors: Luigi Naldini (Milan, IT); Brian Brown (Milan, IT)
Assignees: OSPEDALE SAN RAFFAELE S.R.L.; FONDAZIONE TELETHON
C12N15/1137C12N15/635C12N15/86A61K48/00C12N2310/141C12N2740/15043C12N2740/15045C12N2760/20222C12N2810/6081
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Quick Facts
Patent No.
US 10,000,757
App. No.
11/921,140
Granted
Jun 19, 2018
Kind
B2
Abstract

A gene vector comprising a miRNA sequence target.

Claims (19)

1. A method comprising delivering a gene vector that comprises at least one miRNA target sequence operably linked to a transgene to:

(i) a cell that comprises a corresponding miRNA,

wherein in said cell expression of the transgene is prevented or reduced; and

(ii) a cell that does not comprise a corresponding miRNA, wherein in said cell the transgene is expressed,

wherein the transgene is a therapeutic gene.

2. A method comprising delivering a gene vector that comprises a transgene operably linked to at least one miRNA target sequence to:

(i) a cell that comprises a corresponding miRNA, thereby preventing immune mediated rejection of the transgene; and

(ii) a cell that does not comprise a corresponding miRNA, wherein in said cell the transgene is expressed, wherein the transgene is a therapeutic gene.

3. A method for preventing immune-mediated rejection according to claim 2 wherein the transferred gene is a circulating antigen.

4. A method of controlling transgene expression comprising delivering a gene vector that comprises at least one miRNA target sequence operably linked to the transgene to a cell that comprises a corresponding miRNA, wherein in said cell the transgene expression is prevented or reduced, and wherein the transgene is a therapeutic gene.

5. The method of claim 4 , wherein the method further comprises delivering the gene vector to a cell that does not comprise a corresponding miRNA, wherein in said cell the transgene is expressed.

6. The method of claim 4 , wherein the prevention or reduction of transgene expression in the cell comprising the corresponding miRNA prevents or reduces a detrimental effect to a subject caused by expression of the transgene in said cell.

7. The method of claim 6 , wherein the detrimental effect is an immune response to the transgene.

8. A method of de-targeting transgene expression from a type of cell in a subject comprising delivering a gene vector that comprises at least one miRNA target sequence operably linked to the transgene to the subject, wherein upon transduction of a cell that comprises a corresponding miRNA by the gene vector, expression of the transgene is prevented or reduced in said cell, and wherein the transgene is a therapeutic gene.

9. The method of claim 8 , wherein upon transduction of a cell that does not comprise a corresponding miRNA by the gene vector, the transgene is expressed in said cell.

10. The method of claim 8 , wherein the prevention or reduction of transgene expression in the cell comprising the corresponding miRNA prevents or reduces a detrimental effect to the subject caused by expression of the transgene in said cell.

11. The method of claim 10 , wherein the detrimental effect is an immune response to the transgene.

12. A method of controlling transgene expression comprising delivering a gene vector that comprises at least one miRNA target sequence operably linked to a transgene to

a cell that comprises a corresponding miRNA wherein in said cell expression of the transgene is prevented or reduced; and wherein the transgene is a toxic or suicide gene.

Assignments (2)
TRANSFER OF BUSINESS Recorded Feb 5, 2013
From: FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR
To: OSPEDALE SAN RAFFAELE S.R.L.
Reel/Frame 029752/0277 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded May 11, 2009
From: NALDINI, LUIGI; BROWN, BRIAN DAVID
To: FONDAZIONE CENTRO SAN RAFFAELE DEL MONTE TABOR; FONDAZIONE TELETHON
Reel/Frame 022666/0575 →
Continuity (2)
Provisional Application 60684954 · May 27, 2005
Related Publication 20100041737A1 · Feb 18, 2010
Cited By (4)
US 12,208,126 US 12,281,321 US 12,516,350 US 12,692,516