IP Library Granted Patent US 10,301,360
Granted Patent B2
US 10,301,360 · App. 15/422,259 · Granted May 28, 2019

Selective recovery

Inventors: Benjamin E. Deverman (Pasadena, CA); Paul H. Patterson (Altadena, CA); Viviana Gradinaru (La Canada-Flintridge, CA)
Assignee: CALIFORNIA INSTITUTE OF TECHNOLOGY
C07K14/005A61K38/1709A61K38/2093A61K38/47A61K38/4813A61K38/50A61K39/3955A61K48/005A61K48/0058C07K7/06C12N7/00C12N15/1068C12N15/86A61K38/00C07K2319/33C12N2750/14122C12N2750/14143C12N2750/14145C12N2810/6027C12Y304/14009C12Y305/01015
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Quick Facts
Patent No.
US 10,301,360
App. No.
15/422,259
Granted
May 28, 2019
Kind
B2
Abstract

Provided herein are methods of selective screening. In addition, various targeting proteins and sequences, as well as methods of their use, are also provided.

Claims (29)

1. A method of generating an AAV with a desired tropism, the method comprising:

providing a population of rAAV genomes, wherein each of the rAAV genomes comprises:

an AAV capsid gene, and

one or more recognition sequences for a Cre recombinase, wherein the one or more recognition sequences are configured to allow generation of a recombinase-dependent change that is detectable, and wherein the one or more recombinase recognition sequences comprises two Cre-recognition sites;

transfecting a target cell expressing the Cre recombinase with the population of rAAV genomes, whereby the Cre recombinase induces a recombination event to generate a recombinase-dependent change in at least one of the rAAV genomes of the population, and wherein the recombinase-dependent change comprises an inversion of the sequences that are flanked by the Cre-recognition sites;

detecting at least one recombinase-dependent change; and

identifying an rAAV genome generated by the at least one recombinase-dependent change, wherein said identified rAAV genome comprises the inversion, and wherein said identified rAAV genome encodes an AAV capsid with the desired tropism.

2. The method of claim 1 , wherein the target cell is present in a tissue, an organ, an organism, or a combination thereof.

3. The method of claim 1 , wherein the capsid genes of the population of rAAV genomes encode mutant capsid proteins.

4. The method of claim 1 , wherein the two Cre-recombinase recognition sites are two loxP or variants of loxP sites in a head-to-head orientation.

5. The method of claim 1 , wherein the target cell is a neuronal cell, a neural stem cell, an astrocytes, a tumor cell, a hematopoetic stem cell, an insulin producing beta cell, a lung epithelium, a skeletal cell, or a cardiac muscle cell.

6. The method of claim 1 , wherein the target cell is located in a brain or spinal cord.

7. The method of claim 1 , further comprising recovering the rAAV that is identified to have a capsid with the desired tropism.

8. A method of generating an AAV with a desired tropism, the method comprising:

providing a population of rAAV genomes, wherein each of the rAAV genomes comprises:

an AAV capsid gene, and

one or more recognition sequences for a recombinase, wherein the one or more recognition sequences are configured to allow generation of a recombinase-dependent change that is detectable;

transfecting a target cell expressing the recombinase with the population of rAAV genomes, wherein the target cell is in a Cre-transgenic mouse, and whereby the recombinase induces a recombination event to generate a recombinase-dependent change in at least one of the rAAV genomes of the population;

detecting at least one recombinase-dependent change; and

identifying an rAAV genome generated by the at least one recombinase-dependent change wherein said identified rAAV genome encodes an AAV capsid with the desired tropism.

9. The method of claim 8 , wherein the target cell is present in a tissue, an organ, an organism, or a combination thereof.

10. The method of claim 8 , wherein the capsid genes of the population of rAAV genomes encode mutant capsid proteins.

11. The method of claim 8 , wherein the recombinase is Cre recombinase.

12. The method of claim 11 , wherein the one or more recombinase recognition sequences comprise two Cre-recognition sites.

13. The method of claim 11 , wherein the recombinase-dependent change comprises an inversion of the sequences that are flanked by the Cre-recognition sites; and wherein the method comprises identifying the rAAV genomes that comprise the inversion.

14. The method of claim 11 , wherein the two Cre-recombinase recognition sites are two loxP or variants of loxP sites in a head-to-head orientation.

15. The method of claim 8 , wherein the target cell is a neuronal cell, a neural stem cell, an astrocytes, a tumor cell, a hematopoetic stem cell, an insulin producing beta cell, a lung epithelium, a skeletal cell, or a cardiac muscle cell.

16. The method of claim 8 , wherein the target cell is located in a brain or spinal cord.

17. The method of claim 8 , further comprising recovering the rAAV that is identified to have a capsid with the desired tropism.

Assignments (4)
CONFIRMATORY LICENSE Recorded Feb 28, 2017
From: CALIFORNIA INSTITUTE OF TECHNOLOGY
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 041392/0796 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 21, 2017
From: GRADINARU, VIVIANA
To: CALIFORNIA INSTITUTE OF TECHNOLOGY
Reel/Frame 041316/0693 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Feb 14, 2017
From: DEVERMAN, BENJAMIN E.
To: CALIFORNIA INSTITUTE OF TECHNOLOGY
Reel/Frame 041251/0618 →
PATENT AND COPYRIGHT AGREEMENT Recorded Feb 14, 2017
From: PATTERSON, PAUL H
To: CALIFORNIA INSTITUTE OF TECHNOLOGY
Reel/Frame 041714/0052 →
Continuity (6)
Division 14485024 · Sep 12, 2014
Provisional Application 61877506 · Sep 13, 2013
Provisional Application 61983624 · Apr 24, 2014
Provisional Application 62020658 · Jul 3, 2014
Provisional Application 62034060 · Aug 6, 2014
Related Publication 20170240885A1 · Aug 24, 2017
Cited By (1)
US 12,653,904