公开(公告)号：US20240238326A1

公开(公告)日：2024-07-18

申请号：US18494445

申请日：2023-10-25

Applicant: Biogen MA Inc. ,  Cold Spring Harbor Laboratory

Inventor： C. Frank Bennett ,  Gene Hung ,  Frank Rigo ,  Adrian R. Krainer ,  Yimin Hua ,  Marco A. Passini ,  Lamya Shihabuddin ,  Seng H. Cheng ,  Katherine W. Klinger

IPC: A61K31/7088 ,  C12N15/113 ,  A61K31/712

CPC classification number: A61K31/7088 ,  C12N15/113 ,  A61K31/712 ,  C12N2310/11 ,  C12N2320/33

Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

公开(公告)号：US12031127B2

公开(公告)日：2024-07-09

申请号：US16091913

申请日：2017-04-05

Applicant: Cold Spring Harbor Laboratory

Inventor： Anthony Zador ,  Justus Kebschull

IPC: C12N15/10 ,  C07K14/18 ,  C12N5/0793 ,  C12N15/09 ,  C12Q1/6806 ,  C40B70/00

CPC classification number: C12N15/1065 ,  C07K14/1808 ,  C12N5/0619 ,  C12N15/09 ,  C12N15/10 ,  C12N15/102 ,  C12Q1/6806 ,  C40B70/00 ,  C12N2770/36143 ,  C12Q1/6806 ,  C12Q2522/101 ,  C12Q2525/161 ,  C12Q2535/122 ,  C12Q2563/179 ,  C12Q2565/514 ,  C12N15/102 ,  C12Q2535/122

Abstract: The present invention provides a composition comprising a plurality of labeled neurons, each of which is labeled by an expression construct that encodes a unique barcoded nucleic acid.

公开(公告)号：US20240102010A1

公开(公告)日：2024-03-28

申请号：US18365787

申请日：2023-08-04

Applicant: Cold Spring Harbor Laboratory

Inventor： Gregory J. Hannon ,  Sihem Cheloufi

IPC: C12N15/113 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N15/111 ,  A01K2207/05 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531 ,  C12N2320/30

Abstract: Provided is an improved design of shRNA based on structural mimics of miR-451 precursors. These miR-451 shRNA mimics are channeled through a novel small RNA biogenesis pathway, require AGO2 catalysis and are processed by Drosha but are independent of DICER processing. This miRNA pathway feeds active elements only into Ago2 because of its unique catalytic activity. These data demonstrate that this newly identified small RNA biogenesis pathway can be exploited in vivo to produce active molecules.

公开(公告)号：US11753641B2

公开(公告)日：2023-09-12

申请号：US17170047

申请日：2021-02-08

Applicant: Cold Spring Harbor Laboratory

Inventor： Gregory J. Hannon ,  Sihem Cheloufi

IPC: C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C12N15/11

CPC classification number: C12N15/113 ,  C12N15/111 ,  A01K2207/05 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/531 ,  C12N2320/30

Abstract: Provided is an improved design of shRNA based on structural mimics of miR-451 precursors. These miR-451 shRNA mimics are channeled through a novel small RNA biogenesis pathway, require AGO2 catalysis and are processed by Drosha but are independent of DICER processing. This miRNA pathway feeds active elements only into Ago2 because of its unique catalytic activity. These data demonstrate that this newly identified small RNA biogenesis pathway can be exploited in vivo to produce active molecules.

公开(公告)号：US11739315B2

公开(公告)日：2023-08-29

申请号：US16403420

申请日：2019-05-03

Applicant: Cold Spring Harbor Laboratory

Inventor： Michael H. Wigler ,  Dan Levy ,  Zihua Wang

IPC: C12N15/10 ,  C12Q1/68 ,  C40B40/06 ,  G16B30/10 ,  C12Q1/6806 ,  C12Q1/6809 ,  C12Q1/6855 ,  G16B30/00

CPC classification number: C12N15/1065 ,  C12N15/1093 ,  C12Q1/68 ,  C12Q1/6806 ,  C12Q1/6809 ,  C12Q1/6855 ,  C40B40/06 ,  G16B30/00 ,  G16B30/10 ,  C12Q2535/122 ,  C12N15/1093 ,  C12Q2525/191 ,  C12Q2535/122 ,  C12Q2563/185

Abstract: The present invention, SMASH (Short Multiply Aggregated Sequence Homologies), is a technique designed to pack multiple independent mappings into every read. Specifically, the invention relates to a composition comprising a first mixture of different chimeric genomic nucleic acid fragments, wherein each different fragment in the mixture comprises randomly ligated DNA segments, wherein each DNA segment in the fragment is a nucleic acid molecule at least 27 base pairs in length resulting from random fragmentation of a single genome. The invention also relates to methods for generating said composition and use of said composition to obtain genomic information, for example, copy number variation.

公开(公告)号：US11624074B2

公开(公告)日：2023-04-11

申请号：US14443357

申请日：2013-11-19

Applicant: Cold Spring Harbor Laboratory

Inventor： Zachary Lippman ,  Soon-Ju Park

IPC: C12N15/82 ,  A01H5/08 ,  C07K14/415 ,  A01H6/82 ,  A01H1/02

Abstract: Provided herein are genetically-altered Solanaceae plants, compositions related to the Solanaceae plants, and methods of making the Solanaceae plants.

公开(公告)号：US20220372473A1

公开(公告)日：2022-11-24

申请号：US17553602

申请日：2021-12-16

Applicant: Ionis Pharmaceuticals, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Frank Rigo ,  C. Frank Bennett ,  Adrian R. Krainer ,  Zhenxun Wang

IPC: C12N15/113

Abstract: The present invention provides compounds comprising oligonucleotides complementary to a pyruvate kinase M transcript. Certain such compounds are useful for hybridizing to a pyruvate kinase M transcript, including but not limited to a pyruvate kinase M transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the pyruvate kinase M transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with cancer.

公开(公告)号：US20220118000A1

公开(公告)日：2022-04-21

申请号：US17379793

申请日：2021-07-19

Applicant: Stoke Therapeutics, Inc. ,  Cold Spring Harbor Laboratory

Inventor： Isabel Aznarez ,  Huw M. Nash ,  Adrian Krainer

IPC: A61K31/7125 ,  A61K47/68 ,  C12N15/113 ,  A61P43/00 ,  A61K38/00 ,  A61K38/17 ,  C12N15/62 ,  C12N15/11

Abstract: Provided herein are methods and compositions for increasing the expression of a protein, and for treating a subject in need thereof, e.g., a subject with deficient protein expression or a subject having a disease described herein.

公开(公告)号：US20210290653A1

公开(公告)日：2021-09-23

申请号：US17337762

申请日：2021-06-03

Applicant: Cold Spring Harbor Laboratory ,  Ionis Pharmaceuticals, Inc.

Inventor： Allen Tingjin Yu ,  David L. Spector ,  Frank Rigo ,  Susan M. Freier ,  Jan Bergmann ,  Carmen Berasain

IPC: A61K31/713 ,  A61K9/00 ,  A61K31/7125 ,  C12N15/113

Abstract: Provided herein are methods, compounds, and compositions for reducing expression of lnc05 in a cell or individual. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a cancer in an individual.

公开(公告)号：US20200299706A1

公开(公告)日：2020-09-24

申请号：US16613660

申请日：2018-05-17

Applicant: Cold Spring Harbor Laboratory

Inventor： Zachary Lippman ,  Sebastian Soyk ,  Zachary Hartford Lemmon

IPC: C12N15/82 ,  A01H5/08 ,  A01H5/10

Abstract: Aspects of the disclosure relate to plants, such as Solanaceae plants containing one or more branching events of a mutant Solyc04g005320 gene (or a homolog thereof), a mutant Solycl2g038510 gene (or a homolog thereof), and a mutant Solyc03gl 14840 gene (or a homolog thereof), as well as methods of producing such plants. In some aspects, such plants have one or more improved traits, such as modified inflorescence architecture, modified flower number, modified fruit number, higher yield, higher quality products, and higher fruit productivity.