MX385339B - Vectores aav mejorados adicionales producidos en células de insecto. - Google Patents

Vectores aav mejorados adicionales producidos en células de insecto.

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Publication number
MX385339B
MX385339B MX2016011585A MX2016011585A MX385339B MX 385339 B MX385339 B MX 385339B MX 2016011585 A MX2016011585 A MX 2016011585A MX 2016011585 A MX2016011585 A MX 2016011585A MX 385339 B MX385339 B MX 385339B
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MX
Mexico
Prior art keywords
amino acid
nucleotide sequence
adeno
insect cells
aav
Prior art date
Application number
MX2016011585A
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English (en)
Other versions
MX2016011585A (es
Inventor
Harald Peter Albert Petry
Jacek Lubelski
Sebastiaan Menno Bosma
Wilhelmus Theodorus Johannes Maria Christiaan Hermens
Original Assignee
Uniqure Ip Bv
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Filing date
Publication date
Application filed by Uniqure Ip Bv filed Critical Uniqure Ip Bv
Publication of MX2016011585A publication Critical patent/MX2016011585A/es
Publication of MX385339B publication Critical patent/MX385339B/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/026—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a baculovirus
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
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  • General Health & Medical Sciences (AREA)
  • Virology (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente invención se refiere a la producción de vectores víricos asociados a adeno en las células de insecto. Las células de insecto por lo tanto comprenden una primera secuencia de nucleótidos que codifica las proteínas de la cápside del virus asociado a adeno (AAV), mediante lo cual el codón de inicio para la traducción de la proteína de la cápside AAV VP1 es un codón de inicio subóptimo, no ATG, y en donde la secuencia de codificación para uno o más residuos de aminoácidos se ha insertado entre el codón de inicio de traducción subóptimo y el codón que codifica el residuo de aminoácido que corresponde al residuo de aminoácido en la posición 2 de la secuencia de aminoácido de la cápside de tipo natural de la cual el primer residuo de aminoácido es alanina, glicina, valina, ácido aspártico o ácido glutámico. La célula de insecto comprende además una segunda secuencia de nucleótidos que comprende al menos una secuencia de nucleótidos de repetición terminal invertida (ITR) AAV; una tercera secuencia de nucleótidos que comprende una secuencia de codificación Rep52 o Rep40 operablemente ligada a las secuencias de control de expresión para la expresión en una célula de insecto; y, una cuarta secuencia de nucleótidos que comprende una secuencia de codificación Rep78 o Rep68 operablemente ligada a las secuencias de control de expresión para la expresión en la célula de insecto. La invención se refiere adicionalmente a los vectores víricos asociados a adeno con una relación alterada de las proteínas de la cápside víricas.
MX2016011585A 2014-03-10 2015-03-10 Vectores aav mejorados adicionales producidos en células de insecto. MX385339B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP14158610 2014-03-10
PCT/NL2015/050149 WO2015137802A1 (en) 2014-03-10 2015-03-10 Further improved aav vectors produced in insect cells

Publications (2)

Publication Number Publication Date
MX2016011585A MX2016011585A (es) 2016-11-29
MX385339B true MX385339B (es) 2025-03-18

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Family Applications (1)

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MX2016011585A MX385339B (es) 2014-03-10 2015-03-10 Vectores aav mejorados adicionales producidos en células de insecto.

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US (2) US10837027B2 (es)
EP (2) EP4450630A3 (es)
JP (2) JP6683397B2 (es)
KR (1) KR102572449B1 (es)
CN (1) CN106459984B (es)
AU (1) AU2015230094B2 (es)
BR (1) BR112016020783A2 (es)
CA (1) CA2942289C (es)
DK (1) DK3117005T3 (es)
EA (1) EA201691809A1 (es)
ES (1) ES2990174T3 (es)
FI (1) FI3117005T3 (es)
HU (1) HUE068339T2 (es)
IL (1) IL247729B2 (es)
MX (1) MX385339B (es)
PL (1) PL3117005T3 (es)
PT (1) PT3117005T (es)
UA (1) UA120923C2 (es)
WO (1) WO2015137802A1 (es)
ZA (1) ZA201606552B (es)

Families Citing this family (71)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
KR20170096998A (ko) 2014-11-05 2017-08-25 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 aadc 폴리뉴클레오티드
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
WO2016077689A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
AU2017248840B2 (en) * 2016-04-16 2022-07-28 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
BR112018073472A2 (pt) 2016-05-18 2019-08-27 Voyager Therapeutics Inc composições e métodos de tratamento da doença de huntington
US11698377B2 (en) * 2016-08-15 2023-07-11 Genzyme Corporation Methods for detecting AAV
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT
JP2019531787A (ja) 2016-08-30 2019-11-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018204786A1 (en) 2017-05-05 2018-11-08 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
IL271957B2 (en) 2017-07-10 2024-02-01 Uniqure Ip Bv Means and methods for AAV-based gene therapy
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3655538A1 (en) * 2017-07-20 2020-05-27 uniQure IP B.V. Improved aav capsid production in insect cells
EP3662060A2 (en) 2017-08-03 2020-06-10 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3075656A1 (en) 2017-09-29 2019-04-04 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111527201A (zh) * 2017-12-29 2020-08-11 优尼科Ip有限公司 修饰的病毒载体及其制备和使用方法
GB201800903D0 (en) * 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
JP2021523914A (ja) 2018-05-15 2021-09-09 ボイジャー セラピューティクス インコーポレイテッドＶｏｙａｇｅｒ Ｔｈｅｒａｐｅｕｔｉｃｓ，Ｉｎｃ． パーキンソン病を治療するための組成物および方法
US12060567B2 (en) 2018-06-13 2024-08-13 Voyager Therapeutics, Inc. Engineered untranslated regions (UTR) for AAV production
JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法
US11702673B2 (en) 2018-10-18 2023-07-18 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
WO2020104435A1 (en) 2018-11-19 2020-05-28 Uniqure Ip B.V. A companion diagnostic to monitor the effects of gene therapy
AU2019382824A1 (en) 2018-11-19 2021-06-03 Uniqure Ip B.V. Method and means to deliver miRNA to target cells
WO2020104480A1 (en) 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
US20220064671A1 (en) 2019-01-18 2022-03-03 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
GB201901571D0 (en) 2019-02-05 2019-03-27 Oxford Genetics Ltd Inducible AAV sysyem
AU2020221312A1 (en) * 2019-02-15 2021-10-07 Generation Bio Co. Modulation of rep protein activity in closed-ended dna (cedna) production
CN111084888B (zh) * 2019-03-15 2021-12-28 北京锦篮基因科技有限公司 一种用于治疗严重高甘油三酯血症的基因药物
CA3132447A1 (en) * 2019-04-24 2020-10-29 Takara Bio Inc. Aav mutant having brain-targeting property
WO2020223236A1 (en) * 2019-04-29 2020-11-05 The Trustees Of The University Of Pennsylvania Novel aav capsids and compositions containing same
KR20220011664A (ko) * 2019-05-24 2022-01-28 리제너론 파마슈티칼스 인코포레이티드 변형된 바이러스 입자 및 이의 용도
JP2022538217A (ja) * 2019-06-27 2022-09-01 エックス－ケム インコーポレイテッド 昆虫細胞及び哺乳動物細胞におけるタンパク質発現のための組換え導入ベクター
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
WO2021053018A1 (en) 2019-09-16 2021-03-25 Uniqure Ip B.V. Targeting misspliced transcripts in genetic disorders
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
WO2021195491A2 (en) * 2020-03-26 2021-09-30 Asklepios Biopharmaceutical, Inc. Inducible promoter for viral vector production
EP4127146A1 (en) 2020-04-02 2023-02-08 uniQure biopharma B.V. Novel cell line
CA3169087A1 (en) 2020-04-02 2021-10-07 David Johannes Francois DU PLESSIS Dual bifunctional vectors for aav production
US20230374540A1 (en) * 2020-06-25 2023-11-23 Ferring Ventures Sa Improved adeno-associated virus gene therapy vectors
EP4305157A1 (en) 2021-03-09 2024-01-17 Huidagene Therapeutics (Singapore) Pte. Ltd. Engineered crispr/cas13 system and uses thereof
EP4314258A1 (en) 2021-04-02 2024-02-07 uniQure biopharma B.V. Methods for producing single insect cell clones
WO2022253955A2 (en) 2021-06-02 2022-12-08 Uniqure Biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins
AU2022285138A1 (en) 2021-06-02 2023-11-30 Uniqure Biopharma B.V. Adeno-associated virus vectors modified to bind high-density lipoprotein
CN117836010A (zh) 2021-06-21 2024-04-05 优尼科生物制药有限公司 改进的裂解程序
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
EP4392434A1 (en) 2021-08-26 2024-07-03 uniQure biopharma B.V. Insect cell-produced high potency aav vectors with cns-tropism
CN114703203B (zh) * 2022-02-11 2024-08-06 上海渤因生物科技有限公司 杆状病毒载体及其用途
US20250230437A1 (en) 2022-04-12 2025-07-17 Uniqure Biopharma B.V. Novel systems for nucleic acid regulation
US20250297252A1 (en) 2022-04-12 2025-09-25 Uniqure Biopharma B.V. Nucleic acid regulation of SNCA
JP2025513066A (ja) 2022-04-12 2025-04-22 ユニキュアー バイオファーマ ビー．ブイ． Ａｐｏｅの核酸制御
WO2023198702A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
WO2024078584A1 (zh) * 2022-10-13 2024-04-18 康霖生物科技（杭州）有限公司 腺相关病毒的衣壳蛋白编码基因改造方法
WO2024196965A1 (en) * 2023-03-23 2024-09-26 Carbon Biosciences, Inc. Parvovirus compositions and related methods for gene therapy
US12383615B2 (en) 2023-03-23 2025-08-12 Carbon Biosciences, Inc. Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods
WO2024218192A1 (en) 2023-04-18 2024-10-24 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
AU2024256392A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Gene delivery vehicles comprising rna and antibodies
WO2024261220A1 (en) 2023-06-23 2024-12-26 Uniqure Biopharma B.V. Novel fragile x constructs
WO2025051805A1 (en) 2023-09-04 2025-03-13 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles
WO2025078606A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Further novel systems for nucleic acid regulation
WO2025078598A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Novel nucleic acid regulation of huntingtin gene
WO2025078602A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Nucleic acid regulation of msh3 in repeat expansion disorders
WO2025114524A1 (en) 2023-11-30 2025-06-05 Uniqure Biopharma B.V. Formulations for viral drug products
WO2025219443A1 (en) 2024-04-19 2025-10-23 Uniqure Biopharma B.V. Nucleic acid for a1at regulation

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4745051A (en) 1983-05-27 1988-05-17 The Texas A&M University System Method for producing a recombinant baculovirus expression vector
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
ATE404217T1 (de) 1999-06-24 2008-08-15 Univ British Columbia Therapie mit lipoproteinlipase (lpl) variant
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
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
AU2003212708A1 (en) 2002-03-05 2003-09-16 Stichting Voor De Technische Wetenschappen Baculovirus expression system
EP3910063A1 (en) 2003-09-30 2021-11-17 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
DK2438931T3 (da) 2004-09-22 2013-12-02 St Jude Childrens Res Hospital Forbedret ekspression af faktor ix i genterapivektorer
PL1945779T3 (pl) * 2005-10-20 2013-08-30 Uniqure Ip Bv Ulepszone wektory AAV produkowane w komórkach owadzich
CA2655957C (en) * 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
PT2173888T (pt) * 2007-07-26 2016-11-17 Uniqure Ip Bv Resumo
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors

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