JP2022001064A5 - - Google Patents

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JP2022001064A5
JP2022001064A5 JP2021165270A JP2021165270A JP2022001064A5 JP 2022001064 A5 JP2022001064 A5 JP 2022001064A5 JP 2021165270 A JP2021165270 A JP 2021165270A JP 2021165270 A JP2021165270 A JP 2021165270A JP 2022001064 A5 JP2022001064 A5 JP 2022001064A5
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seq
aav
capsid protein
region
acid sequence
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JP2021165270A
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JP2022001064A (ja
JP7395550B2 (ja
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Publication of JP2022001064A5 publication Critical patent/JP2022001064A5/ja
Priority to JP2023060936A priority patent/JP2023076606A/ja
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JP2021165270A 2016-07-26 2021-10-07 新規アデノ随伴ウイルスキャプシドタンパク質 Active JP7395550B2 (ja)

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JP2021215368A JP7303287B2 (ja) 2016-07-26 2021-12-29 新規アデノ随伴ウイルスキャプシドタンパク質
JP2023060936A JP2023076606A (ja) 2016-07-26 2023-04-04 新規アデノ随伴ウイルスキャプシドタンパク質

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US201662366838P 2016-07-26 2016-07-26
US62/366,838 2016-07-26
JP2019503445A JP6994018B2 (ja) 2016-07-26 2017-07-25 新規アデノ随伴ウイルスキャプシドタンパク質

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JP2021215368A Division JP7303287B2 (ja) 2016-07-26 2021-12-29 新規アデノ随伴ウイルスキャプシドタンパク質
JP2023060936A Division JP2023076606A (ja) 2016-07-26 2023-04-04 新規アデノ随伴ウイルスキャプシドタンパク質

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JP2022001064A JP2022001064A (ja) 2022-01-06
JP2022001064A5 true JP2022001064A5 (enExample) 2022-01-13
JP7395550B2 JP7395550B2 (ja) 2023-12-11

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JP2019503445A Expired - Fee Related JP6994018B2 (ja) 2016-07-26 2017-07-25 新規アデノ随伴ウイルスキャプシドタンパク質
JP2021165270A Active JP7395550B2 (ja) 2016-07-26 2021-10-07 新規アデノ随伴ウイルスキャプシドタンパク質
JP2021215368A Active JP7303287B2 (ja) 2016-07-26 2021-12-29 新規アデノ随伴ウイルスキャプシドタンパク質
JP2023060936A Pending JP2023076606A (ja) 2016-07-26 2023-04-04 新規アデノ随伴ウイルスキャプシドタンパク質

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JP2023060936A Pending JP2023076606A (ja) 2016-07-26 2023-04-04 新規アデノ随伴ウイルスキャプシドタンパク質

Country Status (15)

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US (3) US11584780B2 (enExample)
EP (1) EP3491008A2 (enExample)
JP (4) JP6994018B2 (enExample)
KR (2) KR102604510B1 (enExample)
CN (1) CN109715650B (enExample)
AU (2) AU2017301600B2 (enExample)
BR (1) BR112019001532A2 (enExample)
CA (1) CA3031026A1 (enExample)
CL (6) CL2019000167A1 (enExample)
IL (2) IL263801B2 (enExample)
MX (1) MX2019000962A (enExample)
PE (1) PE20190401A1 (enExample)
PH (1) PH12019500068A1 (enExample)
SG (1) SG11201811603WA (enExample)
WO (1) WO2018022608A2 (enExample)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CN108137655B (zh) 2015-09-28 2022-04-22 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
CA3034527A1 (en) 2016-08-23 2018-03-01 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
EP3768695A4 (en) * 2018-02-27 2022-04-06 The Trustees of the University of Pennsylvania NOVEL ADENO-ASSOCIATED VIRUS (AAV) VECTORS, AAV VECTORS WITH REDUCED CAPSIID DEAMIDATION AND USES THEREOF
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
AU2019249890B2 (en) * 2018-04-05 2024-10-10 Association Institut De Myologie Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
EP3790567B1 (en) 2018-05-11 2025-08-06 Massachusetts Eye and Ear Infirmary Liver-specific tropism of adeno-associated viruses
TW202005978A (zh) * 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
EP3841109A4 (en) * 2018-08-21 2022-09-21 Massachusetts Eye and Ear Infirmary COMPOSITIONS AND METHODS TO MODULATE THE TRANSDUCTION EFFICIENCY OF ADENO-ASSOCIATED VIRUSES
WO2020117898A1 (en) * 2018-12-05 2020-06-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
CA3125294A1 (en) * 2019-01-23 2020-07-30 University Of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
CA3133453A1 (en) 2019-03-21 2020-09-24 Daniel Mccoy Recombinant adeno-associated virus vectors
WO2020198737A1 (en) * 2019-03-28 2020-10-01 The General Hospital Corporation Engineered adeno-associated (aav) vectors for transgene expression
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
CN113518824B (zh) * 2019-10-16 2024-02-23 上海药明康德新药开发有限公司 新的aav变体
TW202128736A (zh) 2019-10-17 2021-08-01 美商史崔德生物公司 用於治療c型尼曼—匹克病之腺相關病毒載體
JP2023503850A (ja) 2019-11-14 2023-02-01 バイオマリン ファーマシューティカル インコーポレイテッド 肝臓特異的遺伝子療法ベクターによる遺伝性血管浮腫の治療
BR112022016596A2 (pt) 2020-02-21 2022-11-16 Akouos Inc Composições e métodos para o tratamento de debilitação auditiva não associada à idade em um indivíduo humano
JP2023518327A (ja) * 2020-02-25 2023-04-28 チルドレンズ・メディカル・リサーチ・インスティテュート アデノ随伴ウイルスカプシドポリペプチドおよびベクター
WO2021183895A1 (en) 2020-03-13 2021-09-16 Biomarin Pharmaceutical Inc. Treatment of fabry disease with aav gene therapy vectors
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas
BR112022021635A2 (pt) 2020-04-29 2022-12-06 Bristol Myers Squibb Co Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas
WO2021236908A2 (en) 2020-05-20 2021-11-25 Biomarin Pharmaceutical Inc. Use of regulatory proteins for the production of adeno-associated virus
US20230242905A1 (en) * 2020-07-03 2023-08-03 Genethon Method for Engineering Novel Hybrid AAV Capsids Through Hyper Variable Regions Swapping
CN116745409A (zh) 2020-08-19 2023-09-12 萨雷普塔治疗公司 用于治疗雷特综合征的腺相关病毒载体
PT4211231T (pt) * 2020-09-10 2024-07-24 Univ Devry Val Dessonne Capsídeo de aav modificado por péptido
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
CA3195553A1 (en) * 2020-10-18 2022-04-21 Qiang Wang Improved adeno-associated virus (aav) vector and uses therefor
KR20230078805A (ko) 2020-11-02 2023-06-02 바이오마린 파머수티컬 인크. 아데노-연관 바이러스의 농축 공정
WO2023034990A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
EP4396203A1 (en) 2021-09-03 2024-07-10 BioMarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240384242A1 (en) 2021-09-03 2024-11-21 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034996A1 (en) 2021-09-03 2023-03-09 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
US20240376495A1 (en) 2021-09-03 2024-11-14 Biomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
WO2023034980A1 (en) 2021-09-03 2023-03-09 Bomarin Pharmaceutical Inc. Aav capsid compositions and methods for delivery
TW202332472A (zh) 2021-10-01 2023-08-16 美商拜奧馬林製藥公司 利用aav基因療法載體進行之遺傳性血管水腫治療及治療性調配物
CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
EP4499154A1 (en) * 2022-03-25 2025-02-05 REGENXBIO Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
TW202417631A (zh) 2022-09-22 2024-05-01 美商拜奧馬林製藥公司 用aav基因治療載體治療心肌病
TW202421788A (zh) 2022-09-22 2024-06-01 美商拜奧馬林製藥公司 用aav基因療法載體治療致心律不整性心肌病
CN115925819B (zh) * 2022-12-30 2023-10-13 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2025051970A2 (en) 2023-09-06 2025-03-13 Dinaqor Ag Methods of treatment of hypertrophic cardiomyopathy with aav gene therapy vectors and therapeutic formulations
WO2025054465A1 (en) * 2023-09-08 2025-03-13 California Institute Of Technology Compositions for transport of therapeutic cargos using miniprotein binders targeting ca-iv
WO2025104321A1 (en) * 2023-11-17 2025-05-22 Genethon Prediction of multiple aav properties by deep learning
WO2025214477A1 (en) 2024-04-12 2025-10-16 Skyline Therapeutics (Shanghai) Co., Ltd. Treatment of genetic cardiomyopathies with aav gene therapy vectors

Family Cites Families (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ATE78293T1 (de) 1983-05-27 1992-08-15 Texas A & M Univ Sys Verfahren zur herstellung eines rekombinanten baculovirus-expressionsvektors.
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
DK518384A (da) 1984-01-31 1985-07-01 Idaho Res Found Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse
US4994371A (en) 1987-08-28 1991-02-19 Davie Earl W DNA preparation of Christmas factor and use of DNA sequences
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
RU2273645C9 (ru) 1994-08-17 2006-11-27 Дзе Рокефеллер Юниверсити Полипептид ожирения (ов)(варианты), его аналог (варианты) и слитый белок (варианты), изолированная молекула нуклеиновой кислоты, молекула днк, рекомбинантный вектор клонирования, рекомбинантный вектор экспрессии, фармацевтическая композиция, моноклональное и поликлональное антитело
US6309853B1 (en) 1994-08-17 2001-10-30 The Rockfeller University Modulators of body weight, corresponding nucleic acids and proteins, and diagnostic and therapeutic uses thereof
EP0933997A4 (en) 1996-09-06 2000-08-16 Chiron Corp METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1999003496A1 (en) 1997-07-21 1999-01-28 The University Of North Carolina At Chapel Hill Factor ix antihemophilic factor with increased clotting activity
WO1999054440A1 (en) 1998-04-21 1999-10-28 Micromet Gesellschaft Für Biomedizinische Forschung Mbh CD19xCD3 SPECIFIC POLYPEPTIDES AND USES THEREOF
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
CA2470711C (en) 2001-10-16 2011-07-12 National Institute Of Advanced Industrial Science And Technology Novel n-acetylglucosamine transferase, nucleic acid encoding the same, antibody against the same and use thereof for diagnosing cancer or tumor
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
DK2573170T3 (en) 2001-12-17 2018-04-09 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 9, vectors containing them, and their use
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
US20030198620A1 (en) 2002-04-16 2003-10-23 Keiya Ozawa Method of treating amino acid metabolic disorders using recombinant adeno-associated virus virions
AU2003237159A1 (en) 2002-04-30 2003-11-17 University Of Florida Treatment for phenylketonuria
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
EP1776364A2 (en) 2003-11-17 2007-04-25 BioMarin Pharmaceutical Inc. Processes for preparing tetrahydrobiopterin, and analogs of tetrahydrobiopterin
EP1771571A2 (en) 2004-07-30 2007-04-11 Targeted Genetics Corporation Recombinant aav based vaccine methods
US8003126B2 (en) 2004-11-17 2011-08-23 Biomarin Pharmaceutical Inc. Stable tablet formulation
EP2359865B1 (en) * 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7531341B1 (en) 2006-06-12 2009-05-12 Biomarin Pharmaceutical Inc. Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof
US7534595B2 (en) 2006-06-12 2009-05-19 Biomarin Pharmaceutical Inc. Compositions of prokaryotic phenylalanine ammonia-lyase and methods of using compositions thereof
US7892824B2 (en) 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
AR066354A1 (es) 2007-05-01 2009-08-12 Genzyme Corp La terapia sistematica con factor-1 de crecimiento de tipo insulina reduce la neuropatia periferica diabetica y mejora la funcion renal en la nefropatia diabetica
DK2173888T3 (en) * 2007-07-26 2016-11-28 Uniqure Ip Bv Baculovirus vectors comprising repeating coding sequences WITH differential preferred codons
US7537923B2 (en) 2007-08-17 2009-05-26 Biomarin Pharmaceutical Inc. Compositions of prokaryotic phenylalanine ammonia-lyase and methods of treating cancer using compositions thereof
WO2009088979A1 (en) 2008-01-07 2009-07-16 Biomarin Pharmaceutical Inc. Method of synthesizing tetrahydrobiopterin
WO2009091912A2 (en) 2008-01-15 2009-07-23 Abbott Laboratories Improved mammalian expression vectors and uses thereof
US7943379B2 (en) 2008-04-30 2011-05-17 Nationwide Children's Hospital, Inc. Production of rAAV in vero cells using particular adenovirus helpers
US20110201022A1 (en) 2008-07-30 2011-08-18 Biomarin Pharmaceutical Inc. Assays for detection of phenylalanine ammonia-lyase and antibodies to phenylalanine ammonia-lyase
ES2985035T3 (es) 2008-09-15 2024-11-04 Uniqure Biopharma B V Mutante de polipéptido de factor IX, sus usos y su procedimiento de producción
WO2010127097A1 (en) * 2009-04-30 2010-11-04 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
TWI688395B (zh) 2010-03-23 2020-03-21 英翠克頌公司 條件性表現治療性蛋白質之載體、包含該載體之宿主細胞及彼等之用途
SG183929A1 (en) 2010-03-29 2012-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
FR2977562B1 (fr) 2011-07-06 2016-12-23 Gaztransport Et Technigaz Cuve etanche et thermiquement isolante integree dans une structure porteuse
CA2864879C (en) 2012-02-17 2021-07-20 The Children's Hospital Of Philadelphia Aav vector compositions and methods for gene transfer to cells, organs and tissues
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
HRP20201063T1 (hr) 2013-03-15 2020-11-27 The Trustees Of The University Of Pennsylvania Pripravci za liječenje mpsi
ES2897508T3 (es) * 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
AU2014318890B2 (en) 2013-09-12 2019-03-07 Biomarin Pharmaceutical Inc. Adeno-associated virus Factor VIII vectors
CN115141258B (zh) * 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
CA2939950C (en) 2014-03-09 2023-08-22 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
EP2960336A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies
CN114146063A (zh) 2014-07-02 2022-03-08 川斯勒佰尔公司 信使rna的包封
EP3194600B1 (en) 2014-07-26 2019-08-28 Consiglio Nazionale Delle Ricerche Compositions and methods for treatment of muscular dystrophy
KR20230067694A (ko) 2014-09-24 2023-05-16 시티 오브 호프 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
JP7166168B2 (ja) * 2015-07-30 2022-11-07 マサチューセッツ アイ アンド イヤー インファーマリー 祖先ウイルス配列およびその使用
TWI841908B (zh) 2015-09-24 2024-05-11 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物
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
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
PE20231949A1 (es) 2015-10-30 2023-12-05 Spark Therapeutics Inc VARIANTES DEL FACTOR VIII REDUCIDO CON CpG, COMPOSICIONES Y METODOS Y USOS PARA EL TRATAMIENTO DE TRASTORNOS DE LA HEMOSTASIA
ES2981519T3 (es) 2016-11-04 2024-10-09 Takeda Pharmaceuticals Co Formulaciones de virus adeno-asociados
CN110325199A (zh) 2016-12-30 2019-10-11 宾夕法尼亚州立大学托管会 用于治疗苯丙酮尿症的基因疗法
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
BR112020015511A2 (pt) 2018-02-01 2021-01-26 Homology Medicines, Inc. composições de vírus adeno-associado para transferência de genes da pah e métodos de uso das mesmas
WO2019217513A2 (en) 2018-05-09 2019-11-14 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
JP2021523198A (ja) 2018-05-14 2021-09-02 ビオマリン プハルマセウトイカル インコーポレイテッド 幼若対象におけるaavベクターの安定的発現
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
MX2021012564A (es) 2019-04-19 2022-01-18 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados.
EP3969061A1 (en) 2019-05-14 2022-03-23 BioMarin Pharmaceutical Inc. Methods of redosing gene therapy vectors
AR122409A1 (es) 2020-04-03 2022-09-07 Biomarin Pharm Inc Tratamiento de la fenilcetonuria con aav y formulaciones terapéuticas

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