US2017065640A1PendingUtilityA1

In Vivo and Ex Vivo Expansion of Hematopoietic Stem Cells With a Targeted Combination of Clinically Tested, FDA Approved Drugs

53
Assignee: UNIV PENNSYLVANIAPriority: Apr 12, 2010Filed: Sep 23, 2016Published: Mar 9, 2017
Est. expiryApr 12, 2030(~3.7 yrs left)· nominal 20-yr term from priority
Inventors:Peter S. KleinJian Huang
A61P 35/00A61P 7/06A61P 35/02C12N 2501/415A61K 35/28C12N 5/0647C12N 2510/00C12N 2501/04A61K 45/06A61K 2035/124A61P 19/08C12N 2501/20C12N 2500/12C12N 2501/727C12N 2500/30
53
PatentIndex Score
0
Cited by
0
References
0
Claims

Abstract

The present invention provides a therapeutic approach to maintain and expand HSCs in vivo using currently available medications that target GSK-3 and mTOR. The present invention also provides a system and method for the ex vivo culturing of HSCs, where an mTOR inhibitor is combined with a GSK-3 inhibitor within the culturing conditions.

Claims

exact text as granted — not AI-modified
What is claimed: 
     
         1 . An isolated HSC prepared by a method of culturing the HSC in a medium comprising at least one GSK-3 inhibitor and at least one mTOR inhibitor, wherein the multipotentiality of the isolated HSC is retained during the culturing. 
     
     
         2 . The isolated HSC of  claim 1 , wherein the HSC is derived from a human. 
     
     
         3 . The isolated HSC of  claim 1 , wherein exogenous genetic material has been introduced into the HSC. 
     
     
         4 . The isolated HSC of  claim 1 , wherein the at least one GSK-3 inhibitor is selected from the group consisting of: lithium, LiCl, 6BIO, CHIR-911, DW21, AR-A014418, and TDZD. 
     
     
         5 . The isolated HSC of  claim 1 , wherein the at least one mTOR inhibitor is selected from the group consisting of: rapamycin, temsirolimus, and everolimus. 
     
     
         6 . The isolated HSC of  claim 1 , wherein the medium further comprises at least one cytokine. 
     
     
         7 . The isolated HSC of  claim 1 , wherein the medium further comprises at least one promotion factor. 
     
     
         8 . The isolated HSC of  claim 1 , wherein the HSC is cultured ex vivo. 
     
     
         9 . A culturing medium for expanding and maintaining HSC, comprising at least one GSK-3 inhibitor and at least one mTOR inhibitor, wherein the multipotentiality of the HSC is retained while the HSC is maintained or expanded in the culturing medium. 
     
     
         10 . The culturing medium of  claim 9 , wherein the at least one GSK-3 inhibitor is selected from the group consisting of: lithium, LiCl, 6BIO, CHIR-911, DW21, AR-A014418, and TDZD. 
     
     
         11 . The culturing medium of  claim 9 , wherein the at least one mTOR inhibitor is selected from the group consisting of: rapamycin, temsirolimus, and everolimus. 
     
     
         12 . The culturing medium of  claim 9 , wherein the medium further comprises at least one cytokine. 
     
     
         13 . The culturing medium of  claim 9 , wherein the medium further comprises at least one promotion factor. 
     
     
         14 . A method for the in vivo maintenance and expansion of HSC in a mammal, the method comprising transplanting the HSC into a tissue of the mammal and administering to the mammal at least one GSK-3 inhibitor and at least one mTOR inhibitor. 
     
     
         15 . The method of  claim 10 , wherein the HSC is derived from a human. 
     
     
         16 . The method of  claim 10 , wherein the mammal is a human. 
     
     
         17 . The method of  claim 10 , wherein exogenous genetic material has been introduced into the HSC. 
     
     
         18 . The method of  claim 10 , wherein the at least one GSK-3 inhibitor is selected from the group consisting of: lithium, LiCl, 6BIO, CHIR-911, DW21, AR-A014418, and TDZD. 
     
     
         19 . The method of  claim 10 , wherein the at least one mTOR inhibitor is selected from the group consisting of: rapamycin, temsirolimus, and everolimus. 
     
     
         20 . The method of  claim 10 , wherein the HSC is transplanted in the form of a heterogeneous mixture of cells.

Join the waitlist — get patent alerts

Track US2017065640A1 — get alerts on status changes and closely related new filings.

We store only your email — no account needed. See our privacy policy.