公开(公告)号：US20160244767A1

公开(公告)日：2016-08-25

申请号：US15045999

申请日：2016-02-17

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Michelle L. HASTINGS

IPC: 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.

Abstract translation: 本公开一般涉及包含与囊性纤维化跨膜传导调节子（CFTR）RNA转录物互补的寡核苷酸的化合物。 某些这样的化合物可用于与CFTR RNA转录物杂交，包括但不限于细胞中的CFTR RNA转录物。 在某些实施方案中，这种杂交导致调节CFTR转录物的剪接。 在某些实施方案中，这些化合物用于治疗与囊性纤维化相关的一种或多种症状。

公开(公告)号：US20160024038A1

公开(公告)日：2016-01-28

申请号：US14716138

申请日：2015-05-19

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Grace E. Stutzmann ,  Russell Dahl ,  Christopher H. Kaiho

IPC: C07D307/54 ,  C07D307/52 ,  C07D405/12

CPC classification number: C07D307/54 ,  C07D307/52 ,  C07D405/12 ,  C07D407/12

Abstract: Disclosed herein are methods and compositions comprising compounds capable of normalizing neuronal calcium dyshomeostasis. Also disclosed are methods comprising these compounds for treating neuronal or neurological disorders, including Alzheimer's disease, Parkinson's disease, Huntington's disease, fronto-temporal dementia, Pick's disease, chronic traumatic encepholopathy, traumatic brain injury, stroke, cerebellar ataxia, multiple sclerosis, Down syndrome, and aging-related CNS disorders.

Abstract translation: 本文公开了包含能够使神经元钙发育不稳定性归一化的化合物的方法和组合物。 还公开了包括这些化合物用于治疗神经元或神经障碍的方法，包括阿尔茨海默病，帕金森病，亨廷顿舞蹈病，颞叶性痴呆，皮克病，慢性创伤性脑病，创伤性脑损伤，中风，小脑共济失调，多发性硬化症，唐氏综合征 ，和老化相关的中枢神经系统疾病。

公开(公告)号：US20150315590A1

公开(公告)日：2015-11-05

申请号：US14705579

申请日：2015-05-06

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：US20140275112A1

公开(公告)日：2014-09-18

申请号：US14213614

申请日：2014-03-14

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Grace E. Stutzmann ,  Russell Dahl

IPC: C07D307/52 ,  C07D307/54 ,  C07D407/12 ,  C07D405/12

CPC classification number: C07D307/52 ,  A61K31/341 ,  A61K31/4166 ,  A61K31/4427 ,  C07D307/54 ,  C07D405/12 ,  C07D407/12

Abstract: Disclosed herein are methods and compositions comprising compounds capable of normalizing neuronal calcium dyshomeostasis. Also disclosed are methods comprising these compounds for treating neuronal or neurological disorders, including Alzheimer's disease, Parkinson's disease, Huntington's disease, fronto-temporal dementia, Pick's disease, chronic traumatic encepholopathy, traumatic brain injury, stroke, cerebellar ataxia, multiple sclerosis, Down syndrome, and aging-related CNS disorders.

Abstract translation: 本文公开了包含能够使神经元钙发育不稳定性归一化的化合物的方法和组合物。 还公开了包括这些化合物用于治疗神经元或神经障碍的方法，包括阿尔茨海默病，帕金森病，亨廷顿舞蹈病，颞叶性痴呆，皮克病，慢性创伤性脑病，创伤性脑损伤，中风，小脑共济失调，多发性硬化症，唐氏综合征 ，和老化相关的中枢神经系统疾病。

公开(公告)号：US20250064397A1

公开(公告)日：2025-02-27

申请号：US18944999

申请日：2024-11-12

Applicant: ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE

Inventor： Holly HUNSBERGER

IPC: A61B5/00 ,  A61B5/0205

Abstract: Provided herein are systems, methods and apparatuses for a miniaturized electronic device worn around the subject tracks health data for diagnosis and preclinical studies.

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：US11786507B2

公开(公告)日：2023-10-17

申请号：US17547472

申请日：2021-12-10

Applicant: Rosalind Franklin University of Medicine and Science

Inventor： Johnny He ,  Xiaojie Zhao

IPC: A61K31/04 ,  A61K31/506 ,  A61K45/06 ,  A61P25/00 ,  A61K31/41

CPC classification number: A61K31/41 ,  A61K31/506 ,  A61K45/06 ,  A61P25/00

Abstract: In the present disclosure, doxycycline-inducible astrocyte-specific HIV Tat transgenic mice (iTat), a surrogate HAND model, were treated with PNU-125096, a positive allosteric modulator of α7 nicotinic acetylcholine receptor (α7 nAChR) and effects on Tat-induced behavioral impairments and neuropathologies were observed. This disclosure shows that PNU-125096 treatment significantly improved locomotor, learning and memory deficits of iTat mice while inhibited glial activation and increased PSD-95 expression in the cortex and hippocampus of iTat mice. α7 nAChR knockout eliminated the protective effects of PNU-125096 on iTat mice. In addition, inhibition of p38 phosphorylation by SB239063, a p38 MAPK-specific inhibitor, exacerbated Tat neurotoxicity in iTat mice. These findings demonstrated for the first time that α7 nAChR activation led to protection against HAND and suggest that α7 nAChR and PNU-125096 hold significant promise for development of therapeutics for HAND.

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.

公开(公告)号：US20190309304A1

公开(公告)日：2019-10-10

申请号：US16438015

申请日：2019-06-11

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

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.