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公开(公告)号:US11298343B2
公开(公告)日:2022-04-12
申请号:US16758986
申请日:2018-10-26
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Inventor: Mitchell J. Weiss , Mondira Kundu , Christophe Lechauve
IPC: A61K31/7052 , A61K31/495 , A61K31/4164 , A61P7/06 , A61K31/436 , A61K45/06
Abstract: Methods for removing excess free α-globin in erythroid cells and treating a thalassemia using an agent that activates Unc-51 Like autophagy activating Kinase (ULK) are described.
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92.
公开(公告)号:US11268158B2
公开(公告)日:2022-03-08
申请号:US15136298
申请日:2016-04-22
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Inventor: Brian P. Sorrentino , Sheng Zhou
IPC: C12Q1/70
Abstract: The invention is directed to methods of assessing the safety of therapeutic compounds and therapeutic genetic manipulations, including integrating gene therapy vectors and genome editing. In particular, the invention provides a method, wherein the oncogenic potential of therapeutic compounds and therapeutic genetic manipulations, including integrating gene therapy vectors and genome editing, is determined by determining the percentage of differentiation blocked hematopoietic progenitor cells.
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公开(公告)号:US20210388012A1
公开(公告)日:2021-12-16
申请号:US17288493
申请日:2018-11-05
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Inventor: Richard E. LEE , Jiuyu LIU
IPC: C07F9/6561 , C07D493/04 , A61K45/06
Abstract: In one aspect, the disclosure relates to substituted spectinomycin analogs, including substituted aminomethyl spectinomycin analogs and substituted spectinamide analogs, with increased tolerability and safety, including improved tolerability to parenteral administration. The present disclosure further pertains to methods of making disclosed compounds, pharmaceutical compositions comprising the disclosed compounds, and methods of treating microbial infections using the disclosed compounds, including methods of treating antibiotic resistant infections and tuberculosis. This abstract is intended as a scanning tool for pur-poses of searching in the particular art and is not intended to be limiting of the present disclosure.
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公开(公告)号:US20210299118A1
公开(公告)日:2021-09-30
申请号:US17315136
申请日:2021-05-07
Applicant: St. Jude Children's Research Hospital
Inventor: Heather Smallwood , Marie Morfouace , Martine F. Roussel , Paul G. Thomas
IPC: A61K31/4745 , A61K31/415 , A61K31/427 , A61K31/433 , A61K31/505 , A61K31/519 , A61K31/34 , A61K31/4164 , A61K31/454 , A61K31/277 , A61K31/352 , A61K31/365 , A61K31/416 , A61K31/4174 , A61K31/4439 , A61K31/555 , A61K31/7004 , A61K45/06
Abstract: The invention generally relates to methods of treating viral infections using known drugs and pharmaceutical compositions comprising same. More specifically, the disclosed methods are useful for the treatment of viral infections that are enveloped viruses. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.
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公开(公告)号:US11103597B2
公开(公告)日:2021-08-31
申请号:US15573684
申请日:2016-05-13
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC.
Inventor: John T. Gray
IPC: C12N15/86 , A61K48/00 , A61K35/76 , C12N7/00 , C12N15/861
Abstract: The present invention relates to nucleic acid molecules containing spacers that can be packaged into viral particles and methods of producing them. In a first aspect, the invention features a nucleic acid molecule including a first spacer (SSI); a first inverted terminal repeat (ITR1); a cloning site (CS); a second inverted terminal repeat (ITR2); and a second spacer (SS2), such as a eukaryotic spacer; operably linked to each other in a 5′-to-3′ direction as: SS1-ITR1-CS-ITR2-SS2. In an embodiment, the invention features a vector comprising any of the above-described nucleic acid molecules. In another aspect, the invention features a plurality of viral particles including the nucleic acid molecule. The invention further includes a host cell including any of the above-described vectors.
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公开(公告)号:US20210230545A1
公开(公告)日:2021-07-29
申请号:US17258533
申请日:2019-07-08
Applicant: St. Jude Children's Research Hospital
Inventor: Caitlin Zebley , Hossam Abdelsamed , Benjamin Youngblood
IPC: C12N5/0783 , C07K14/725 , C12Q1/6876 , A61K35/17
Abstract: Provided herein are methods and compositions for modulating T-cell activity by incubating a CD8 T cell with a signal 3 cytokine, such as IL-12. Incubation of naïve CD8 T cells, particularly, with a signal 3 cytokine can acquire long-lived memory associated gene expression characteristic of the stem cell memory subset of CD8 T cells. Further, incubation with signal 3 cytokines can induce changes to the epigenetic profile of naïve CD8 T cells that are more characteristic of bona fide Tscm cells than in vitro generated cells using traditional differentiation protocols. On account of epigenetic profiles being preserved during in vivo homeostasis, signal 3 cytokines such as IL-12 can be used to engineer a T cell population with the desired epigenetic profile that maintains effector functions and proliferative capacity.
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公开(公告)号:US20210188777A1
公开(公告)日:2021-06-24
申请号:US17192683
申请日:2021-03-04
Applicant: St. Jude Children's Research Hospital
Inventor: R. Kip Guy , P. Jake Slavish , William Robert Shadrick , Brandon M. Young , Vincent A. Boyd , Nagakumar Bharatham , Jeanine E. Price , Anang Shelat
IPC: C07D215/42 , C07D215/44 , C07D215/46 , C07D401/10 , C07D401/12 , C07D405/10 , C07D405/12 , C07D409/10 , C07D413/12
Abstract: In one aspect, compounds and compositions that modulate a bromodomain and methods of making and using same are disclosed. The disclosed compounds and compositions can be useful for disorders associated with inhibition of a bromodomain such as, for example, cancer. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.
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98.
公开(公告)号:US20210179676A1
公开(公告)日:2021-06-17
申请号:US16758905
申请日:2018-10-26
Applicant: ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Inventor: J. Paul Taylor , Peipei Zhang
IPC: C07K14/415 , C12N9/14
Abstract: A nucleic acid molecule encoding a fusion protein composed of an inducible multimerization moiety at the amino terminus, and a GTPase-Activating Protein SH3 Domain-Binding Protein (G3BP) is provided for exogenous stimulus/G3BP-mediated stress granule formation. Further disclosed are chemical or light inducible multimerization proteins or protein domains that can be used for the nucleic acid molecule production, and a method of inducing stress granule formation in a cell comprising expressing the nucleic acid molecule.
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99.
公开(公告)号:US20210164041A1
公开(公告)日:2021-06-03
申请号:US17166549
申请日:2021-02-03
Applicant: St. Jude Children's Research Hospital, Inc.
Inventor: Shengdar Q. Tsai , Cicera R. Lazzarotto
IPC: C12Q1/6874 , C12N15/10
Abstract: The invention relates to a high-throughput method for characterizing the genome-wide activity of editing nucleases in vitro.
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100.
公开(公告)号:US10920272B2
公开(公告)日:2021-02-16
申请号:US16695719
申请日:2019-11-26
Applicant: St. Jude Children's Research Hospital, Inc.
Inventor: Shengdar Q. Tsai , Cicera R. Lazzarotto
IPC: C12Q1/6874 , C12N15/10 , C40B50/06
Abstract: The invention relates to a high-throughput method for characterizing the genome-wide activity of editing nucleases in vitro.
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