公开(公告)号：WO2021216778A2

公开(公告)日：2021-10-28

申请号：PCT/US2021/028489

申请日：2021-04-21

Applicant: ALLEN INSTITUTE

Inventor： DAIGLE, Tanya ,  GRAYBUCK, Lucas T. ,  KALMBACH, Brian Edward ,  LEIN, Edward Sebastian ,  LEVI, Boaz P. ,  MICH, John K. ,  SEDENO CORTES, Adriana Estela ,  TASIC, Bosiljka ,  TING, Jonathan ,  ZENG, Hongkui

IPC: C12N15/67 ,  C12N15/864 ,  C12N15/63 ,  C12N15/86 ,  C12N2750/14143 ,  C12N2830/00 ,  C12N2830/008

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. Particularly, the artificial expression constructs can be used to selectively express synthetic genes and / or modify gene expression in neocortical glutamatergic layer 5 neurons, such as glutamatergic layer 5 extratelencephalic-projecting (L5 ET) neurons.

公开(公告)号：WO2022103859A1

公开(公告)日：2022-05-19

申请号：PCT/US2021/058812

申请日：2021-11-10

Applicant: ALLEN INSTITUTE

Inventor： DAIGLE, Tanya ,  GRAYBUCK, Lucas T. ,  KALMBACH, Brian Edward ,  LEIN, Edward Sebastian ,  LEVI, Boaz P. ,  MICH, John K. ,  SEDENO CORTES, Adriana Estela ,  TASIC, Bosiljka ,  TING, Jonathan ,  ZENG, Hongkui

IPC: C12N15/86 ,  C12N15/861

Abstract: Artificial expression constructs for modulating gene expression in targeted central nervous system cell types are described. The artificial expression constructs can be used to express synthetic genes or modify gene expression in chandelier cells. Chandelier cells are a subtype of GABAergic interneurons that that have been implicated in disorders such as epilepsy and schizophrenia.

公开(公告)号：WO2023060112A1

公开(公告)日：2023-04-13

申请号：PCT/US2022/077586

申请日：2022-10-05

Applicant: ALLEN INSTITUTE

Inventor： DAIGLE, Tanya ,  LEIN, Edward Sebastian ,  LEVI, Boaz P. ,  TASIC, Bosiljka ,  TING, Jonathan ,  ZENG, Hongkui ,  MICH, John K.

IPC: C12N15/63 ,  C12N15/67 ,  A61K35/76

Abstract: Artificial expression constructs for modulating gene expression in targeted central nervous system cell types are described. The artificial expression constructs can be used to express synthetic genes or modify gene expression in a cerebellar cell type and a secondary cell type.

公开(公告)号：WO2021248085A2

公开(公告)日：2021-12-09

申请号：PCT/US2021/036028

申请日：2021-06-04

Applicant: ALLEN INSTITUTE

Inventor： DAIGLE, Tanya ,  GRAYBUCK, Lucas T. ,  LEIN, Sebastian ,  LEVI, Boaz P. ,  MICH, John K. ,  SEDENO CORTES, Adriana Estela ,  TASIC, Bosiljka ,  TING, Jonathan ,  WALKER, Miranda ,  ZENG, Hongkui

IPC: C12N15/09 ,  C12N15/11 ,  C12N15/63 ,  C12N15/67 ,  A61K48/0058 ,  C12N2750/14143

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in inhibitory neocortical GABAergic neurons including somatostatin GABAergic neurons, parvalbumin GABAergic neurons, vasointestinal peptide GABAergic neurons, Lamp5 GABAergic neurons, and in some instances astrocytes.

公开(公告)号：WO2020168279A3

公开(公告)日：2020-08-20

申请号：PCT/US2020/018416

申请日：2020-02-14

Applicant: ALLEN INSTITUTE

Inventor： TING, Jonathan ,  LEVI, Boaz P. ,  TASIC, Bosiljka ,  MICH, John K. ,  HESS, Erik ,  LEIN, Edward Sebastian ,  GRAYBUCK, Lucas T. ,  DAIGLE, Tanya ,  ZENG, Hongkui

IPC: A61K48/00 ,  C12N15/09 ,  C12N15/11 ,  C12N15/63 ,  C12N15/67 ,  C12N15/85

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in GABAergic neurons generally; and/or GABAergic neuron cell types such as lysosomal associated membrane protein 5 (Lamp5) neurons; vasoactive intestinal polypeptide-expressing (Vip) neurons; somatostatin (Sst) neurons; and/or parvalbumin (Pvalb) neuron cell types. Certain artificial expression constructs additionally drive selective gene expression in Layer 4 and/or layer 5 intratelencephalic (IT) neurons, deep cerebellar nuclear neurons or cerebellar Purkinje cells.

公开(公告)号：WO2020097121A1

公开(公告)日：2020-05-14

申请号：PCT/US2019/059927

申请日：2019-11-05

Applicant: ALLEN INSTITUTE

Inventor： GRAYBUCK, Lucas T. ,  TASIC, Bosiljka ,  DAIGLE, Tanya ,  TING, Jonathan ,  ZENG, Hongkui ,  KALMBACH, Brian Edward ,  MICH, John K. ,  HESS, Erik ,  LEIN, Edward Sebastian ,  LEVI, Boaz P.

IPC: C12N15/67 ,  C12N15/85 ,  C12N5/0793 ,  A61K48/00

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in excitatory cortical neurons, such as primarily within cortical layers 2/3, 4, 5, and 6 and including those with extratelencephalic (ET) projections, intratelencephalic (IT) projections, and pyramidal tract (PT) projections, among others.

公开(公告)号：WO2022246276A2

公开(公告)日：2022-11-24

申请号：PCT/US2022/030371

申请日：2022-05-20

Applicant: ALLEN INSTITUTE

Inventor： DAIGLE, Tanya ,  LEIN, Edward Sebastian ,  LEVI, Boaz P. ,  MICH, John K. ,  MITCHELL, Amanda ,  TASIC, Bosiljka ,  TING, Jonathan ,  WEED, Natalie ,  ZENG, Hongkui

IPC: C12N15/79 ,  C12N15/86 ,  G01N33/543

Abstract: Artificial expression constructs for modulating gene expression in targeted central nervous system cell types are described. The artificial expression constructs can be used to express synthetic genes or modify gene expression in GABAergic or glutamatergic neurons within the thalamus. In some cases, the artificial constructs can also be used to express synthetic genes or modify gene expression in neurons within the thalamus as well as a secondary cell type.

公开(公告)号：WO2022036255A1

公开(公告)日：2022-02-17

申请号：PCT/US2021/045995

申请日：2021-08-13

Applicant: ALLEN INSTITUTE

Inventor： DAIGLE, Tanya ,  LEIN, Edward Sebastian ,  LEVI, Boaz P. ,  MICH, John K. ,  TASIC, Bosiljka ,  TING, Jonathan ,  ZENG, Hongkui

IPC: C12N15/11 ,  C12N15/63 ,  C12N15/67 ,  C12N15/85

Abstract: Artificial expression constructs for modulating gene expression in striatal neurons are described. The artificial expression constructs can be used to express heterologous genes in striatal neurons including in striatal medium spiny neuron-pan, striatal medium spiny neuron-indirect pathway, striatal medium spiny neuron-direct pathway, striatal interneuron-cholinergic, and Drd3+ medium spiny neurons in olfactory tubercle. The artificial expression constructs can be used for many purposes, including to research and treat movement disorders such as Parkinson's disease and Huntington's disease.

公开(公告)号：WO2020168279A2

公开(公告)日：2020-08-20

申请号：PCT/US2020/018416

申请日：2020-02-14

Applicant: ALLEN INSTITUTE

Inventor： TING, Jonathan ,  LEVI, Boaz P. ,  TASIC, Bosiljka ,  MICH, John K. ,  HESS, Erik ,  LEIN, Edward Sebastian ,  GRAYBUCK, Lucas T. ,  DAIGLE, Tanya ,  ZENG, Hongkui

IPC: A01K67/027

Abstract: Artificial expression constructs for selectively modulating gene expression in selected central nervous system cell types are described. The artificial expression constructs can be used to selectively express synthetic genes or modify gene expression in GABAergic neurons generally; and/or GABAergic neuron cell types such as lysosomal associated membrane protein 5 (Lamp5) neurons; vasoactive intestinal polypeptide-expressing (Vip) neurons; somatostatin (Sst) neurons; and/or parvalbumin (Pvalb) neuron cell types. Certain artificial expression constructs additionally drive selective gene expression in Layer 4 and/or layer 5 intratelencephalic (IT) neurons, deep cerebellar nuclear neurons or cerebellar Purkinje cells.