KR20160147974A - 망막 및 cns 유전자 요법을 위한 aav 벡터 - Google Patents

망막 및 cns 유전자 요법을 위한 aav 벡터 Download PDF

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KR20160147974A
KR20160147974A KR1020167033392A KR20167033392A KR20160147974A KR 20160147974 A KR20160147974 A KR 20160147974A KR 1020167033392 A KR1020167033392 A KR 1020167033392A KR 20167033392 A KR20167033392 A KR 20167033392A KR 20160147974 A KR20160147974 A KR 20160147974A
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amino acid
raav
nucleic acid
numbering
substitution
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Korean (ko)
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아브라함 스카리아
제니퍼 설리반
리사 엠. 스타넥
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젠자임 코포레이션
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Application filed by 젠자임 코포레이션 filed Critical 젠자임 코포레이션
Priority to KR1020247006248A priority Critical patent/KR102863726B1/ko
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KR1020167033392A 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터 Ceased KR20160147974A (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020247006248A KR102863726B1 (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US61/988,131 2014-05-02
US201562114575P 2015-02-10 2015-02-10
US62/114,575 2015-02-10
PCT/US2015/028966 WO2015168666A2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Related Child Applications (1)

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KR1020247006248A Division KR102863726B1 (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터

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KR20160147974A true KR20160147974A (ko) 2016-12-23

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KR1020167033392A Ceased KR20160147974A (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터
KR1020247006248A Active KR102863726B1 (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터
KR1020257031397A Pending KR20250141850A (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터

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KR1020247006248A Active KR102863726B1 (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터
KR1020257031397A Pending KR20250141850A (ko) 2014-05-02 2015-05-02 망막 및 cns 유전자 요법을 위한 aav 벡터

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US (3) US10982228B2 (enExample)
EP (3) EP3913061A1 (enExample)
JP (4) JP6741591B2 (enExample)
KR (3) KR20160147974A (enExample)
CN (2) CN114231563B (enExample)
AU (3) AU2015252797C1 (enExample)
CA (1) CA2946593A1 (enExample)
CL (2) CL2016002713A1 (enExample)
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DO (1) DOP2016000280A (enExample)
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FI (1) FI3137497T4 (enExample)
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HU (1) HUE054768T2 (enExample)
IL (2) IL292951B2 (enExample)
LT (1) LT3137497T (enExample)
MX (2) MX2016014220A (enExample)
MY (1) MY188987A (enExample)
PE (1) PE20170260A1 (enExample)
PH (1) PH12016502046A1 (enExample)
PL (1) PL3137497T5 (enExample)
PT (1) PT3137497T (enExample)
RS (1) RS62078B2 (enExample)
SG (3) SG10201809739QA (enExample)
SI (1) SI3137497T2 (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20200098481A (ko) * 2017-10-16 2020-08-20 비게네론 게엠베하 Aav 벡터

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WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
EP2561075B1 (en) 2010-04-23 2018-06-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
WO2011133890A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
WO2011133874A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Multicistronic expression constructs
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
ES2661680T3 (es) 2011-04-21 2018-04-03 University Of Massachusetts Composiciones basadas en VAAr y métodos para tratar deficiencias de alfa-1 anti-tripsina
HRP20220036T1 (hr) 2011-04-22 2022-04-01 The Regents Of The University Of California Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe
KR20240093939A (ko) * 2013-05-15 2024-06-24 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
EP3561062A1 (en) 2013-09-13 2019-10-30 California Institute of Technology Selective recovery
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
JP6741591B2 (ja) 2014-05-02 2020-08-19 ジェンザイム・コーポレーション 網膜およびcns遺伝子治療用aavベクター
RU2718248C2 (ru) * 2014-05-14 2020-03-31 Эстев Фармасьютикалс, С.А. Аденоассоциированные вирусные векторы для лечения лизосомных болезней накопления
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるaavベクター
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
HUE057272T2 (hu) * 2015-02-10 2022-04-28 Genzyme Corp Vírusrészecskék fokozott bevitele a csíkolt testbe és az agykéregbe
EA201791805A1 (ru) 2015-02-10 2018-05-31 Джензим Корпорейшн ВАРИАНТ СРЕДСТВА ДЛЯ RNAi
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
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