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    KCC2 expression enhancing compounds and uses thereof
    43.
    发明授权
    KCC2 expression enhancing compounds and uses thereof 有权

    公开(公告)号：US12053465B2

    公开(公告)日：2024-08-06

    申请号：US17713880

    申请日：2022-04-05

    Applicant: Whitehead Institute for Biomedical Research

    Inventor： Rudolf Jaenisch ,  Xin Tang

    IPC: A61K31/496 ,  A61K31/05 ,  A61K31/404 ,  A61K31/4525 ,  A61K31/47 ,  A61K31/4709 ,  A61K31/519 ,  A61P25/28

    CPC classification number: A61K31/496 ,  A61K31/05 ,  A61K31/404 ,  A61K31/4525 ,  A61K31/47 ,  A61K31/4709 ,  A61K31/519 ,  A61P25/28

    Abstract: Potassium chloride cotransporter-2 (KCC2) plays a critical role in brain function, and deficiency in KCC2 has been linked to neurological diseases, psychiatric disorders, and central nervous system injuries. In particular, Rett syndrome (RTT), a severe neurodevelopmental disorder caused by mutations in the X-linked gene Methyl CpG binding Protein 2 (MECP2), has been linked to deficits in KCC2. The disclosure reports the use of CRISPR/Cas9 genome-editing technology to generate stem cell-derived, genetically defined KCC2 reporter human neurons for large-scale compound screening. This screening platform has been utilized to identify a number of small molecule compounds that are capable of enhancing KCC2 expression in both wild-type and RTT neurons, as well as organotypical brain slices cultured from wild-type mice. These first-in class compounds may be applied as a novel therapeutic approach to restore the impaired balance between excitation and inhibition observed in neurological diseases, psychiatric disorders, and central nervous system injuries.

    KCC2 expression enhancing compounds and uses thereof
    46.
    发明授权
    KCC2 expression enhancing compounds and uses thereof 有权

    公开(公告)号：US11331313B2

    公开(公告)日：2022-05-17

    申请号：US16615676

    申请日：2018-05-22

    Applicant: Whitehead Institute for Biomedical Research

    Inventor： Rudolf Jaenisch ,  Xin Tang

    IPC: A61K31/496 ,  A61P25/28 ,  A61K31/05 ,  A61K31/404 ,  A61K31/4525 ,  A61K31/47 ,  A61K31/4709 ,  A61K31/519

    Abstract: Potassium chloride cotransporter-2 (KCC2) plays a critical role in brain function, and deficiency in KCC2 has been linked to neurological diseases, psychiatric disorders, and central nervous system injuries. In particular, Rett syndrome (RTT), a severe neurodevelopmental disorder caused by mutations in the X-linked gene Methyl CpG binding Protein 2 (MECP2), has been linked to deficits in KCC2. The disclosure reports the use of CRISPR/Cas9 genome-editing technology to generate stem cell-derived, genetically defined KCC2 reporter human neurons for large-scale compound screening. This screening platform has been utilized to identify a number of small molecule compounds that are capable of enhancing KCC2 expression in both wild-type and RTT neurons, as well as organotypical brain slices cultured from wild-type mice. These first-in class compounds may be applied as a novel therapeutic approach to restore the impaired balance between excitation and inhibition observed in neurological diseases, psychiatric disorders, and central nervous system injuries.

    REPORTER OF GENOMIC METHYLATION AND USES THEREOF
    47.
    发明申请
    REPORTER OF GENOMIC METHYLATION AND USES THEREOF 审中-公开

    公开(公告)号：US20190194764A1

    公开(公告)日：2019-06-27

    申请号：US16032975

    申请日：2018-07-11

    Applicant: Whitehead Institute for Biomedical Research

    Inventor： Yonatan Stelzer ,  Rudolf Jaenisch

    IPC: C12Q1/6897 ,  G01N33/50 ,  C12Q1/68 ,  C12N15/90 ,  C07K14/65 ,  A61K48/00

    CPC classification number: C12Q1/6897 ,  A61K48/00 ,  C07K14/65 ,  C12N15/907 ,  C12N2310/10 ,  C12N2310/20 ,  C12Q1/68 ,  G01N33/5023

    Abstract: In some aspects, described herein is a DNA methylation reporter. In some aspects, the DNA methylation reporter comprises a promoter whose activity can be affected by exogenous methylation changes without being independently regulated by the DNA methylation machinery, operably linked to a DNA sequence that encodes a reporter molecule. In some embodiments the DNA methylation reporter comprises (i) a promoter derived from a mammalian imprinted gene promoter; and (ii) a sequence that encodes a reporter molecule that is detectable in individual mammalian cells, wherein the promoter is operably linked to the sequence that encodes the reporter molecule. Also described are nucleic acids that comprise the DNA methylation reporter, cells that have the DNA methylation reporter integrated into their genome, and non-human mammals comprising cells that have the DNA methylation reporter integrated into their genome. Also described are methods of measuring DNA methylation of a region of interest located in proximity to the DNA methylation reporter in the genome of a cell by detecting the reporter molecule.

    Reporter of genomic methylation and uses thereof
    48.
    发明授权
    Reporter of genomic methylation and uses thereof 有权

    公开(公告)号：US10023922B2

    公开(公告)日：2018-07-17

    申请号：US15078851

    申请日：2016-03-23

    Applicant: Whitehead Institute for Biomedical Research

    Inventor： Yonatan Stelzer ,  Rudolf Jaenisch

    IPC: C12Q1/68 ,  G01N33/50 ,  C12Q1/6897 ,  A61K48/00 ,  C07K14/65

    Abstract: In some aspects, described herein is a DNA methylation reporter. In some aspects, the DNA methylation reporter comprises a promoter whose activity can be affected by exogenous methylation changes without being independently regulated by the DNA methylation machinery, operably linked to a DNA sequence that encodes a reporter molecule. In some embodiments the DNA methylation reporter comprises (i) a promoter derived from a mammalian imprinted gene promoter; and (ii) a sequence that encodes a reporter molecule that is detectable in individual mammalian cells, wherein the promoter is operably linked to the sequence that encodes the reporter molecule. Also described are nucleic acids that comprise the DNA methylation reporter, cells that have the DNA methylation reporter integrated into their genome, and non-human mammals comprising cells that have the DNA methylation reporter integrated into their genome. Also described are methods of measuring DNA methylation of a region of interest located in proximity to the DNA methylation reporter in the genome of a cell by detecting the reporter molecule.

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