JP2020533968A5 - - Google Patents

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Publication number
JP2020533968A5
JP2020533968A5 JP2020511199A JP2020511199A JP2020533968A5 JP 2020533968 A5 JP2020533968 A5 JP 2020533968A5 JP 2020511199 A JP2020511199 A JP 2020511199A JP 2020511199 A JP2020511199 A JP 2020511199A JP 2020533968 A5 JP2020533968 A5 JP 2020533968A5
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JP
Japan
Prior art keywords
amino acids
sequence
sequences
seq
raavrec2
Prior art date
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Pending
Application number
JP2020511199A
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English (en)
Japanese (ja)
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JP2020533968A (ja
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Publication date
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Priority claimed from PCT/US2018/047466 external-priority patent/WO2019040586A1/en
Publication of JP2020533968A publication Critical patent/JP2020533968A/ja
Publication of JP2020533968A5 publication Critical patent/JP2020533968A5/ja
Pending legal-status Critical Current

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JP2020511199A 2017-08-25 2018-08-22 組換えアデノ随伴ベクター Pending JP2020533968A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762550458P 2017-08-25 2017-08-25
US62/550,458 2017-08-25
PCT/US2018/047466 WO2019040586A1 (en) 2017-08-25 2018-08-22 RECOMBINANT ADENO-ASSOCIATE VECTORS

Publications (2)

Publication Number Publication Date
JP2020533968A JP2020533968A (ja) 2020-11-26
JP2020533968A5 true JP2020533968A5 (enExample) 2021-09-30

Family

ID=65439655

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020511199A Pending JP2020533968A (ja) 2017-08-25 2018-08-22 組換えアデノ随伴ベクター

Country Status (10)

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US (2) US20190085358A1 (enExample)
EP (1) EP3655041A4 (enExample)
JP (1) JP2020533968A (enExample)
KR (1) KR20200042935A (enExample)
CN (1) CN111163811A (enExample)
AU (1) AU2018320849A1 (enExample)
CA (1) CA3073937A1 (enExample)
IL (1) IL272655A (enExample)
MX (1) MX2020002148A (enExample)
WO (1) WO2019040586A1 (enExample)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020106927A1 (en) 2018-11-21 2020-05-28 Certego Therapeutics Gaboxadol for reducing risk of suicide and rapid relief of depression
CA3149164A1 (en) * 2019-09-03 2021-03-11 Suma Krishnan Compositions and methods for the treatment of congenital ichthyoses
MX2022014599A (es) 2020-05-20 2022-12-16 Certego Therapeutics Inc Gaboxadol deuterado en anillo y su uso para el tratamiento de trastornos psiquiatricos.
CA3191543A1 (en) * 2020-09-04 2022-03-10 Lei Cao Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
WO2023044306A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants

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JP4969002B2 (ja) * 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン rAAV形質導入を増加するための化合物および方法
EP1546369A4 (en) * 2002-08-12 2007-01-17 Univ Michigan DIAGNOSIS AND TREATMENT OF DISEASES THROUGH DEFECTS IN THE TUBEROUS SKLEROSEPFAD
US7427396B2 (en) * 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
EP3085389A1 (en) * 2005-04-07 2016-10-26 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2692868A1 (en) * 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
BR112015027336A2 (pt) * 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
PT3024498T (pt) * 2013-07-22 2020-03-06 Childrens Hospital Philadelphia Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015171547A1 (en) * 2014-05-05 2015-11-12 Ovid Therapeutics Inc. Methods of treating cognitive impairment associated with neurodegenerative disorders
KR20170005494A (ko) * 2014-05-30 2017-01-13 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 잠복 바이러스 감염에 대한 치료제를 전달하는 조성물 및 방법
US10577627B2 (en) * 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3200830B1 (en) * 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
EP3875593A1 (en) * 2015-12-11 2021-09-08 California Institute of Technology Targeting peptides for directing adeno-associated viruses (aavs)
AU2016370630B2 (en) * 2015-12-14 2023-04-13 The Trustees Of The University Of Pennsylvania Adeno-associated viral vectors useful in treatment of spinal muscular atropy
WO2017197355A2 (en) * 2016-05-13 2017-11-16 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
CA3052829A1 (en) * 2017-02-15 2018-08-23 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
CN110621318A (zh) * 2017-03-15 2019-12-27 奥维德医疗公司 排他地由设计者药物激活的设计者受体在治疗癫痫发作紊乱中的用途
MX2020006473A (es) * 2017-12-20 2020-09-22 Ovid Therapeutics Inc Uso de hm4di en el tratamiento de trastornos convulsivos.
WO2019241624A1 (en) * 2018-06-14 2019-12-19 Ovid Therapeutics Inc. Use of mir-92a or mir-145 in the treatment of angelman syndrome
SG11202103102WA (en) * 2018-09-26 2021-04-29 California Inst Of Techn Adeno-associated virus compositions for targeted gene therapy
US10557149B1 (en) * 2019-07-15 2020-02-11 Vigene Biosciences, Inc. Recombinantly-modified adeno-associated virus helper vectors and their use to improve the packaging efficiency of recombinantly-modified adeno-associated virus
EP4069263A4 (en) * 2019-12-05 2024-03-06 The Board Of Regents Of The University Of Texas System TRANSGENIC CASSETTES DESIGNED TO EXPRESS A HUMAN MECP2 GENE
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WO2022235702A1 (en) * 2021-05-04 2022-11-10 California Institute Of Technology Recombinant aavs for delivery to central nervous system and brain vasculature
CN114259502A (zh) * 2021-12-24 2022-04-01 南京鼓楼医院 一种基于脂肪组织的中枢靶向递送siRNA的方法及应用
CN115554418B (zh) * 2022-11-22 2023-04-14 四川至善唯新生物科技有限公司 一种重组腺相关病毒载体的药物组合物及其用途

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