TW202325850A - Aadc、gdnf多核苷酸及其用於治療帕金森病 - Google Patents

Aadc、gdnf多核苷酸及其用於治療帕金森病 Download PDF

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TW202325850A
TW202325850A TW111145716A TW111145716A TW202325850A TW 202325850 A TW202325850 A TW 202325850A TW 111145716 A TW111145716 A TW 111145716A TW 111145716 A TW111145716 A TW 111145716A TW 202325850 A TW202325850 A TW 202325850A
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polynucleotide
polya
seq
sequence
aav
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TW111145716A
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姜軍
吳明月
寧威
廖成
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大陸商上海瑞宏迪醫藥有限公司
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TW111145716A 2021-11-29 2022-11-29 Aadc、gdnf多核苷酸及其用於治療帕金森病 TW202325850A (zh)

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Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023202637A1 (en) 2022-04-19 2023-10-26 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating neurodegenerative disorders
CN118252953A (zh) * 2022-12-26 2024-06-28 科辉智药(深圳)新药研究中心有限公司 一种用于神经退行性疾病治疗的基因序列构建体及其应用
WO2025040166A1 (zh) 2023-08-23 2025-02-27 上海瑞宏迪医药有限公司 药物组合物及其用途
WO2025060451A1 (zh) * 2023-09-18 2025-03-27 上海金珂博生物技术有限公司 经修饰的腺相关病毒载体及其在治疗神经疾病中的用途
CN117904201B (zh) * 2023-10-19 2026-03-27 广州逐鹿生物科技有限公司 一种用于清除乙肝病毒的病毒基因治疗载体及应用
US20250333807A1 (en) * 2024-04-29 2025-10-30 Agathos Biologics Recombinant Adeno-Associated Virus (rAAV) Universal Reference Standard for Determining rAAV Genome Copy Titer by Quantitative PCR

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX9205293A (es) 1991-09-20 1993-05-01 Syntex Sinergen Neuroscience J Factores neurotrofico derivado del glial
US6184200B1 (en) 1995-09-28 2001-02-06 Amgen Inc. Truncated glial cell line-derived neurotrophic factor
US5865744A (en) 1996-09-16 1999-02-02 Lemelson; Jerome H. Method and system for delivering therapeutic agents
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
AU1071701A (en) 1999-09-29 2001-04-30 Trustees Of The University Of Pennsylvania, The Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US20030050273A1 (en) * 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
SI3235827T1 (sl) 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
CN101213305A (zh) * 2004-12-09 2008-07-02 建新公司 转基因在哺乳动物中枢神经系统中的可调控型表达
WO2006110689A2 (en) 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
US20100203083A1 (en) 2007-05-31 2010-08-12 Medigene Ag Mutated structural protein of a parvovirus
FI20070808A0 (fi) 2007-10-25 2007-10-25 Mart Saarma GDNF:n silmukointivariantit ja niiden käytöt
US8340743B2 (en) 2007-11-21 2012-12-25 MRI Interventions, Inc. Methods, systems and computer program products for positioning a guidance apparatus relative to a patient
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
JP5559185B2 (ja) * 2008-11-11 2014-07-23 オックスフォード バイオメディカ(ユーケー)リミテッド 方法
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
WO2013096955A1 (en) 2011-12-23 2013-06-27 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
AU2013221212B2 (en) 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
US9636370B2 (en) 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
JP6396988B2 (ja) 2013-03-15 2018-09-26 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二重グリカン結合性aavベクターのための方法および組成物
US9585971B2 (en) 2013-09-13 2017-03-07 California Institute Of Technology Recombinant AAV capsid protein
WO2015048534A1 (en) 2013-09-26 2015-04-02 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
IL248102B (en) 2014-05-02 2022-07-01 Genzyme Corp aav vectors for gene therapy of the central nervous system and retina
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
BR112017005892A2 (pt) 2014-09-24 2017-12-12 Hope City variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
AU2015335923B2 (en) 2014-10-21 2021-04-29 University Of Massachusetts Recombinant AAV variants and uses thereof
SG11201703148TA (en) * 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
DE202015010000U1 (de) 2014-12-12 2023-07-03 CureVac SE Artifizielle Nukleinsäuremoleküle für eine verbesserte Proteinexpression
TN2017000354A1 (en) 2015-02-10 2019-01-16 Genzyme Corp VARIANT RNAi
CA2977355A1 (en) 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
US10240145B2 (en) 2015-11-25 2019-03-26 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
JOP20190269A1 (ar) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3668980A1 (en) 2017-08-15 2020-06-24 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
CN108841868A (zh) * 2018-05-31 2018-11-20 康霖生物科技(杭州)有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
EP4306643A3 (en) 2018-10-11 2024-03-20 ethris GmbH Plasmid containing a sequence encoding an mrna with a segmented poly(a) tail
KR20210149251A (ko) * 2019-03-08 2021-12-08 옵시디안 테라퓨틱스, 인크. 조율가능한 조절을 위한 인간 탄산 무수화효소 2 조성물 및 방법
CN109971729B (zh) * 2019-04-19 2021-07-16 上海信致医药科技有限公司 一种酶组合物

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US20250051750A1 (en) 2025-02-13
EP4442830A1 (en) 2024-10-09
CA3238867A1 (en) 2023-06-01
KR20240110849A (ko) 2024-07-16
JP2024543918A (ja) 2024-11-26
CN118525098A (zh) 2024-08-20

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