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公开(公告)号:US12042508B2
公开(公告)日:2024-07-23
申请号:US17472046
申请日:2021-09-10
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.
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公开(公告)号:US11786507B2
公开(公告)日:2023-10-17
申请号:US17547472
申请日:2021-12-10
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.
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公开(公告)号:US20230272021A1
公开(公告)日:2023-08-31
申请号:US17991219
申请日:2022-11-21
Inventor: Bruce L. Riser
IPC: C07K14/47 , A61K38/17 , A61K38/04 , A61K9/00 , A61K35/12 , A61K35/28 , A61K35/51 , A61K38/10 , C07K14/475
CPC classification number: C07K14/47 , A61K38/1709 , A61K38/04 , A61K9/0014 , A61K35/12 , A61K35/28 , A61K35/51 , A61K38/10 , C07K14/4743 , C07K14/475
Abstract: The present invention provides a method for treating a human patient with a pathology by administering to the subject an effective amount of an agent selected from the group of: native full-length CCN3 proteins; analog CCN3 full-length proteins with native cysteine residues substituted by a replacement amino acid; CCNp native peptide fragments having from about 12 to about 20 amino acids; analog CCNp peptide fragments with native cysteine residues substituted with a replacement amino acid; and combinations thereof.
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公开(公告)号:US20220054526A1
公开(公告)日:2022-02-24
申请号:US17472046
申请日:2021-09-10
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.
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公开(公告)号:US10472634B2
公开(公告)日:2019-11-12
申请号:US15315828
申请日:2015-06-04
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.
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46.
公开(公告)号:US20190309304A1
公开(公告)日:2019-10-10
申请号:US16438015
申请日:2019-06-11
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.
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公开(公告)号:US10351608B2
公开(公告)日:2019-07-16
申请号:US14821494
申请日:2015-08-07
Inventor: Bruce L. Riser
IPC: C07K14/47 , A61K38/17 , A61K38/04 , A61K9/00 , A61K35/12 , A61K38/10 , C07K14/475 , A61K35/28 , A61K35/51
Abstract: The present invention provides a method for treating a human patient with a pathology by administering to the subject an effective amount of an agent selected from the group of: native full-length CCN3 proteins; analog CCN3 full-length proteins with native cysteine residues substituted by a replacement amino acid; CCNp native peptide fragments having from about 12 to about 20 amino acids; analog CCNp peptide fragments with native cysteine residues substituted with a replacement amino acid; and combinations thereof.
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公开(公告)号:US10011578B2
公开(公告)日:2018-07-03
申请号:US15671833
申请日:2017-08-08
Inventor: Grace E. Stutzmann , Russell Dahl , Christopher H. Kaiho
IPC: C07D307/52 , C07D307/54 , C07D405/12 , C07D407/12
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.
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公开(公告)号:US09827478B1
公开(公告)日:2017-11-28
申请号:US14616522
申请日:2015-02-06
Inventor: Bijan Najafi , James Stanley Wrobel , Ali-Reza Boloori
CPC classification number: A63B69/3608 , A63B71/0619 , A63B2071/0647 , A63B2220/40 , A63B2220/836 , A63B2230/62 , G06F19/3481 , G09B19/0038
Abstract: The present disclosure is directed to a body-worn sensor-based system for evaluating the biomechanics and the motor adaptation characteristics of postural control during a sport activity such as a golf swing. Various embodiments use sensors such as accelerometers, gyroscopes, and magnetometers to measure the three-dimensional motion of ankle and hip joints. In several embodiments, additional sensors attached to other body segments are used to improve the accuracy of the data or detect particular instants during the swing (e.g., top of back swing, instant of the maximum speed of arm, and instant of ball impact). In a golf embodiment, the system combines the measured data in conjunction with a biomechanical model of the human body to: (1) estimate the two-dimensional sway of the golfer's center of mass; (2) quantify and evaluate the golfer's balance via his/her postural compensatory strategy; and (3) provide visual feedback to the golfer for improving dynamic postural control.
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公开(公告)号:US09725429B2
公开(公告)日:2017-08-08
申请号:US14716138
申请日:2015-05-19
Inventor: Grace E. Stutzmann , Russell Dahl , Christopher H. Kaiho
IPC: C07D307/54 , C07D405/12 , C07D307/52 , C07D407/12
CPC classification number: C07D307/54 , C07D307/52 , C07D405/12 , C07D407/12
Abstract: Disclosed herein are methods and compositions comprising compounds of formula (I) 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.
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