CN117813314A - 用于治疗神经纤维瘤疾患的组合物和方法 - Google Patents

用于治疗神经纤维瘤疾患的组合物和方法 Download PDF

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Publication number
CN117813314A
CN117813314A CN202280049047.XA CN202280049047A CN117813314A CN 117813314 A CN117813314 A CN 117813314A CN 202280049047 A CN202280049047 A CN 202280049047A CN 117813314 A CN117813314 A CN 117813314A
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China
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aav
seq
nucleic acid
sequence
merlin
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Pending
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CN202280049047.XA
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English (en)
Chinese (zh)
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S·R·普洛特金
M·伍顿
D·苏海
S·高
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Nf2 Treatment Co
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Nf2 Treatment Co
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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CN202280049047.XA 2021-06-08 2022-06-08 用于治疗神经纤维瘤疾患的组合物和方法 Pending CN117813314A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163202359P 2021-06-08 2021-06-08
US63/202,359 2021-06-08
PCT/US2022/032679 WO2022261209A1 (en) 2021-06-08 2022-06-08 Compositions and methods for treating neurofibromatic disorders

Publications (1)

Publication Number Publication Date
CN117813314A true CN117813314A (zh) 2024-04-02

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CN202280049047.XA Pending CN117813314A (zh) 2021-06-08 2022-06-08 用于治疗神经纤维瘤疾患的组合物和方法

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US (1) US20230056226A1 (https=)
EP (1) EP4352079A4 (https=)
JP (1) JP2024520815A (https=)
CN (1) CN117813314A (https=)
WO (1) WO2022261209A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3216712A1 (en) * 2021-04-14 2022-10-20 Research Institute At Nationwide Children's Hospital Materials and methods for neurofibromin 2/merlin (nf2) gene therapy
WO2025054256A1 (en) * 2023-09-06 2025-03-13 Tenaya Therapeutics, Inc. Targeting peptides, engineered aav capsid proteins and virions
CN118291543A (zh) * 2024-02-29 2024-07-05 神拓生物技术(杭州)有限公司 一种二型神经纤维瘤基因治疗的慢病毒载体及其应用
WO2025205770A1 (ja) * 2024-03-26 2025-10-02 国立大学法人 東京大学 スタッファーｄｎａを含む組換えプラスミド

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP2726079A4 (en) * 2011-06-28 2014-12-17 Glaxosmithkline Ip No 2 Ltd ADMINISTRATIVE AND TREATMENT PROCEDURES
WO2015171270A1 (en) * 2014-05-06 2015-11-12 Felder Mitchell S Method for the treatment of neurofibromatosis
EP3448874A4 (en) * 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
WO2020012149A1 (en) * 2018-07-13 2020-01-16 Ucl Business Ltd Glucocerebrosidase gene therapy

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EP4352079A1 (en) 2024-04-17
US20230056226A1 (en) 2023-02-23
EP4352079A4 (en) 2025-05-07
WO2022261209A1 (en) 2022-12-15
JP2024520815A (ja) 2024-05-24

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