公开(公告)号：US20170137826A1

公开(公告)日：2017-05-18

申请号：US15349840

申请日：2016-11-11

Applicant: Rosalind Franklin University of Medicine and Science ,  The McLean Hospital Corporation

Inventor： Michelle L. Hastings ,  Ole Isacson ,  Joanna A. Korecka-Roet

IPC: C12N15/113

CPC classification number: C12N15/1137 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2320/33 ,  C12Y207/11001 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/322 ,  C12N2310/3533

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript. Certain such compounds are useful for hybridizing to a LRRK2 RNA transcript, including but not limited to a LRRK2 RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRRK2 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Parkinson's disease.

公开(公告)号：US11732260B2

公开(公告)日：2023-08-22

申请号：US16977649

申请日：2019-03-01

Applicant: Ionis Pharmaceuticals, Inc. ,  Rosalind Franklin University of Medicine and Science

Inventor： Frank Rigo ,  Michelle L. Hastings

IPC: C07H21/04 ,  C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/346 ,  C12N2320/33

Abstract: Certain embodiments disclosed herein are directed to compounds and methods for modulating APP expression. In certain embodiments, modulating the splicing of amyloid precursor protein (APP) reduces amyloid β (Aβ) production.

公开(公告)号：US11116785B2

公开(公告)日：2021-09-14

申请号：US16730517

申请日：2019-12-30

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

IPC: A61K31/712 ,  C12N15/113

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：US20140243388A1

公开(公告)日：2014-08-28

申请号：US14176722

申请日：2014-02-10

Applicant: ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

Inventor： Michelle L. Hastings

IPC: C12N15/113

CPC classification number: C12N15/113 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C12N2310/11 ,  C12N2320/33

Abstract: The present invention provides a method for treating Usher's syndrome in a human subject including administering to the human subject an oligonucleotide having 8 to 30 linked nucleosides having a nucleobase sequence comprising a complementary region comprising at least 8 contiguous nucleobases complementary to a target region of equal length within exon 3 of an Usher RNA transcript.

Abstract translation: 本发明提供了一种治疗人类受试者的Usher综合征的方法，包括向人类受试者施用具有8至30个连接核苷的寡核苷酸，所述核苷酸具有包含至少8个连续核苷酸序列的互补区的互补区，该互补区与等长的靶区域互补 在Usher RNA转录本的外显子3内。

公开(公告)号：US10544417B2

公开(公告)日：2020-01-28

申请号：US15835995

申请日：2017-12-08

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

IPC: C12N15/113

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：US10525076B2

公开(公告)日：2020-01-07

申请号：US15835698

申请日：2017-12-08

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

IPC: A61K31/712 ,  C12N15/113

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：US09840709B2

公开(公告)日：2017-12-12

申请号：US15045999

申请日：2016-02-17

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

IPC: C12N15/11 ,  C12N15/113

CPC classification number: C12N15/1137 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/351 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2320/30 ,  C12N2320/33

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：US12042508B2

公开(公告)日：2024-07-23

申请号：US17472046

申请日：2021-09-10

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

IPC: A61K31/712 ,  C12N15/113

CPC classification number: A61K31/712 ,  C12N15/1138 ,  A01K2267/0306 ,  C12N2310/11 ,  C12N2310/321 ,  C12N2310/3233 ,  C12N2320/33

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：US20220054526A1

公开(公告)日：2022-02-24

申请号：US17472046

申请日：2021-09-10

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. Hastings

IPC: A61K31/712 ,  C12N15/113

Abstract: The present disclosure relates generally to compounds comprising oligonucleotides complementary to a cystic fibrosis transmembrane conductance regulator (CFTR) RNA transcript. Certain such compounds are useful for hybridizing to a CFTR RNA transcript, including but not limited to a CFTR RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CFTR transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Cystic Fibrosis.

公开(公告)号：US10472634B2

公开(公告)日：2019-11-12

申请号：US15315828

申请日：2015-06-04

Applicant: Ionis Pharmaceuticals, Inc. ,  Rosalind Franklin University of Medicine and Science

Inventor： Frank Rigo ,  Michelle L. Hastings

IPC: C12N15/113 ,  C12N5/00

Abstract: The present invention provides compounds comprising oligonucleotides complementary to an LRP8 transcript. Certain such compounds are useful for hybridizing to an LRP8 transcript, including but not limited, to an LRP8 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRP8 transcript. In certain embodiments, such hybridization results in an increase in inclusion of exon 19 in the LRP8 mRNA transcript. In certain embodiments, such compounds are used to treat Alzheimer's Disease.