JP2014239686A5 - - Google Patents

Download PDF

Info

Publication number
JP2014239686A5
JP2014239686A5 JP2014144592A JP2014144592A JP2014239686A5 JP 2014239686 A5 JP2014239686 A5 JP 2014239686A5 JP 2014144592 A JP2014144592 A JP 2014144592A JP 2014144592 A JP2014144592 A JP 2014144592A JP 2014239686 A5 JP2014239686 A5 JP 2014239686A5
Authority
JP
Japan
Prior art keywords
amino acid
nucleotide sequence
insect cell
sequence
parvovirus
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2014144592A
Other languages
English (en)
Japanese (ja)
Other versions
JP2014239686A (ja
Filing date
Publication date
Application filed filed Critical
Publication of JP2014239686A publication Critical patent/JP2014239686A/ja
Publication of JP2014239686A5 publication Critical patent/JP2014239686A5/ja
Pending legal-status Critical Current

Links

JP2014144592A 2007-07-26 2014-07-14 差次的コドンバイアスを有する反復コード配列を含むバキュロウイルスベクター Pending JP2014239686A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US95208107P 2007-07-26 2007-07-26
EP07113257 2007-07-26
US60/952,081 2007-07-26
EP07113257.5 2007-07-26

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
JP2010518140A Division JP5634262B2 (ja) 2007-07-26 2008-07-25 差次的コドンバイアスを有する反復コード配列を含むバキュロウイルスベクター

Publications (2)

Publication Number Publication Date
JP2014239686A JP2014239686A (ja) 2014-12-25
JP2014239686A5 true JP2014239686A5 (enExample) 2015-07-09

Family

ID=38753583

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2010518140A Active JP5634262B2 (ja) 2007-07-26 2008-07-25 差次的コドンバイアスを有する反復コード配列を含むバキュロウイルスベクター
JP2014144592A Pending JP2014239686A (ja) 2007-07-26 2014-07-14 差次的コドンバイアスを有する反復コード配列を含むバキュロウイルスベクター

Family Applications Before (1)

Application Number Title Priority Date Filing Date
JP2010518140A Active JP5634262B2 (ja) 2007-07-26 2008-07-25 差次的コドンバイアスを有する反復コード配列を含むバキュロウイルスベクター

Country Status (19)

Country Link
US (5) US8697417B2 (enExample)
EP (3) EP2173888B1 (enExample)
JP (2) JP5634262B2 (enExample)
KR (1) KR101597695B1 (enExample)
CN (1) CN101868547B (enExample)
AU (1) AU2008279883B2 (enExample)
BR (1) BRPI0814459B1 (enExample)
CA (1) CA2694406C (enExample)
DK (2) DK3093345T3 (enExample)
EA (1) EA022169B1 (enExample)
ES (1) ES2602585T3 (enExample)
HU (1) HUE029744T2 (enExample)
IL (1) IL203535A (enExample)
MX (1) MX2010000944A (enExample)
NZ (1) NZ582881A (enExample)
PL (2) PL2173888T3 (enExample)
PT (1) PT2173888T (enExample)
SI (2) SI2173888T1 (enExample)
WO (1) WO2009014445A2 (enExample)

Families Citing this family (109)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SI2173888T1 (sl) * 2007-07-26 2017-01-31 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
IT1400425B1 (it) 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
US20140155469A1 (en) 2011-04-19 2014-06-05 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors and viruses
GB201210357D0 (en) 2012-06-12 2012-07-25 Ucl Business Plc Factor VIII sequences
EA036394B1 (ru) 2013-10-24 2020-11-05 ЮНИКЬЮРЕ АйПи Б.В. Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний
FI3117005T3 (fi) * 2014-03-10 2024-08-29 Uniqure Ip Bv Hyönteissoluissa tuotettuja lisää parannettuja aav-vektoreita
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
BR112017009497A2 (pt) 2014-11-05 2018-02-06 Voyager Therapeutics, Inc. polinucleotídeos de aadc para o tratamento da doença de parkinson
GB201420139D0 (en) 2014-11-12 2014-12-24 Ucl Business Plc Factor IX gene therapy
CA2967367C (en) 2014-11-14 2023-05-16 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US10570395B2 (en) 2014-11-14 2020-02-25 Voyager Therapeutics, Inc. Modulatory polynucleotides
JP6741345B2 (ja) 2014-11-28 2020-08-19 ユニキュアー アイピー ビー.ブイ. パルボウイルスビリオンを含む組成物におけるdna不純物
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
US10174321B2 (en) 2014-12-24 2019-01-08 Uniqure Ip B.V. RNAI induced huntingtin gene suppression
AU2017254652B2 (en) * 2016-04-21 2022-12-01 Virovek, Inc. AAV production in insect cells, methods and compositions therefor
WO2017189959A1 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
RU2764587C2 (ru) 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
JP7066635B2 (ja) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド 調節性ポリヌクレオチド
SG11201811603WA (en) 2016-07-26 2019-02-27 Biomarin Pharm Inc Novel adeno-associated virus capsid proteins
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING DEFICIENCY IN A HUMAN SUBJECT
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
WO2018109207A1 (en) 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
EP3618839A4 (en) 2017-05-05 2021-06-09 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
HUE069232T2 (hu) 2017-07-10 2025-02-28 Uniqure Ip Bv Módok és eljárások AAV génterápiára emberekben
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
AU2018311069A1 (en) 2017-08-03 2020-02-20 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
EP3687582A4 (en) 2017-09-29 2021-07-14 Voyager Therapeutics, Inc. RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2018394287B2 (en) 2017-12-29 2024-09-26 Uniqure Ip B.V. Modified viral vectors and methods of making and using the same
HRP20231126T1 (hr) 2018-01-17 2024-01-05 Meiragtx Uk Ii Limited Modificirani protein kapsida raav za gensku terapiju
GB201800903D0 (en) 2018-01-19 2018-03-07 Oxford Genetics Ltd Vectors
EP3793616A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
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
EP3807404A1 (en) 2018-06-13 2021-04-21 Voyager Therapeutics, Inc. Engineered 5' untranslated regions (5' utr) for aav production
CA3103963A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
WO2020053258A1 (en) 2018-09-12 2020-03-19 Uniqure Ip B.V. Rnai induced c9orf72 suppression for the treatment of als/ftd
WO2020061418A1 (en) * 2018-09-20 2020-03-26 Tallgrass Biologics L.L.C. Treatment and control of colony collapse disorder
CN109295172A (zh) * 2018-09-26 2019-02-01 浙江海洋大学 大黄鱼清道夫受体家族基因的密码子偏好性分析方法
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
AU2019354793A1 (en) 2018-10-05 2021-05-13 Voyager Therapeutics, Inc. Engineered nucleic acid constructs encoding AAV production proteins
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
WO2020081490A1 (en) 2018-10-15 2020-04-23 Voyager Therapeutics, Inc. EXPRESSION VECTORS FOR LARGE-SCALE PRODUCTION OF rAAV IN THE BACULOVIRUS/Sf9 SYSTEM
WO2020104480A1 (en) 2018-11-19 2020-05-28 Uniqure Biopharma B.V. Adeno-associated virus vectors for expressing fviii mimetics and uses thereof
AU2019385638A1 (en) 2018-11-19 2021-06-03 Uniqure Ip B.V. A companion diagnostic to monitor the effects of gene therapy
WO2020104295A1 (en) 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
CA3120177A1 (en) 2018-11-19 2020-05-28 Uniqure Ip B.V. Method and means to deliver mirna to target cells
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
TW202106879A (zh) 2019-04-29 2021-02-16 美商航海家醫療公司 於生物反應器中生產經桿狀病毒感染之昆蟲細胞(biic)之系統及方法
US20220267797A1 (en) 2019-07-15 2022-08-25 Meiragtx Uk Ii Limited Modified AAV Capsid Proteins for Treatment of Arthritic Disease
WO2021030125A1 (en) 2019-08-09 2021-02-18 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
WO2021041375A1 (en) * 2019-08-26 2021-03-04 Rootpath Genomics, Inc. Compositions and methods for producing adeno-associated viral vectors
EP4022070A1 (en) 2019-08-26 2022-07-06 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4031148A1 (en) 2019-09-16 2022-07-27 uniQure IP B.V. Targeting misspliced transcripts in genetic disorders
TW202134440A (zh) * 2019-12-04 2021-09-16 美商聖加莫治療股份有限公司 用於生產重組aav之新穎組合物及方法
WO2021168362A1 (en) 2020-02-21 2021-08-26 Akouos, Inc. Compositions and methods for treating non-age-associated hearing impairment in a human subject
WO2021195491A2 (en) * 2020-03-26 2021-09-30 Asklepios Biopharmaceutical, Inc. Inducible promoter for viral vector production
KR20220163950A (ko) * 2020-04-02 2022-12-12 유니큐어 바이오파마 비.브이. Aav 생산을 위한 이중 이기능성 벡터
AU2021249483A1 (en) 2020-04-02 2022-09-15 Uniqure Biopharma B.V. Novel cell line
JP2023521347A (ja) 2020-04-07 2023-05-24 ユニキュアー アイピー ビー.ブイ. アンジオポエチン様3(angptl3)をサイレンシングするための遺伝子構築物及びその使用
WO2021211753A1 (en) 2020-04-15 2021-10-21 Voyager Therapeutics, Inc. Tau binding compounds
MX2023000815A (es) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composiciones y metodos para el tratamiento de trastornos neurologicos relacionados con la deficiencia de glucosilceramidasa beta.
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
EP4192514A1 (en) 2020-08-06 2023-06-14 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
US20240141377A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115427561B (zh) 2021-03-09 2024-06-04 辉大(上海)生物科技有限公司 工程化CRISPR/Cas13系统及其用途
WO2022214635A1 (en) 2021-04-08 2022-10-13 Stichting Vu Nucleic acid molecules for compensation of stxbp1 haploinsufficiency and their use in the treatment of stxbp1-related disorders
EP4347849A1 (en) 2021-06-02 2024-04-10 uniQure biopharma B.V. Adeno-associated virus vectors modified to bind high-density lipoprotein
EP4347798A2 (en) 2021-06-02 2024-04-10 uniQure biopharma B.V. Insect cell production of parvoviral vectors with modified capsid proteins
US20250382611A1 (en) 2021-06-21 2025-12-18 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
EP4359010A1 (en) 2021-06-21 2024-05-01 uniQure biopharma B.V. Improved lysis procedures
WO2023283962A1 (en) 2021-07-16 2023-01-19 Huigene Therapeutics Co., Ltd. Modified aav capsid for gene therapy and methods thereof
KR20240021951A (ko) * 2021-07-28 2024-02-19 바이오마린 파머수티컬 인크. 대규모 아데노-관련 바이러스 생산 시스템
WO2023025920A1 (en) 2021-08-26 2023-03-02 Uniqure Biopharma B.V. Insect cell-produced high potency aav vectors with cns-tropism
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023147374A2 (en) 2022-01-25 2023-08-03 Voyager Therapeutics, Inc. Baculovirus expression system
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
EP4508216A1 (en) 2022-04-12 2025-02-19 uniQure biopharma B.V. Nucleic acid regulation of snca
WO2023198662A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Novel systems for nucleic acid regulation
WO2023198702A1 (en) 2022-04-12 2023-10-19 Uniqure Biopharma B.V. Nucleic acid regulation of c9orf72
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
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2024054983A1 (en) 2022-09-08 2024-03-14 Voyager Therapeutics, Inc. Controlled expression of viral proteins
EP4644556A1 (en) 2022-12-28 2025-11-05 FUJIFILM Corporation Method for producing adeno-associated virus, and vector set
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
AU2024256392A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Gene delivery vehicles comprising rna and antibodies
AU2024258047A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Novel neurotropic adeno-associated virus capsids with detargeting of peripheral organs
AU2024258816A1 (en) 2023-04-18 2025-10-23 Uniqure Biopharma B.V. Generation of adeno-associated virus capsid libraries for insect cells
TW202449167A (zh) 2023-04-26 2024-12-16 美商航海家醫療公司 用於治療肌肉萎縮性脊髓側索硬化症之組成物及方法
WO2024261220A1 (en) 2023-06-23 2024-12-26 Uniqure Biopharma B.V. Novel fragile x constructs
WO2025049993A1 (en) 2023-08-31 2025-03-06 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025051805A1 (en) 2023-09-04 2025-03-13 Uniqure Biopharma B.V. Novel neurotropic recombinant adeno-associated virus particles
WO2025078602A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Nucleic acid regulation of msh3 in repeat expansion disorders
WO2025078598A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Novel nucleic acid regulation of huntingtin gene
WO2025078606A1 (en) 2023-10-11 2025-04-17 Uniqure Biopharma B.V. Further novel systems for nucleic acid regulation
WO2025114524A1 (en) 2023-11-30 2025-06-05 Uniqure Biopharma B.V. Formulations for viral drug products
WO2025171227A1 (en) 2024-02-08 2025-08-14 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
WO2025219443A1 (en) 2024-04-19 2025-10-23 Uniqure Biopharma B.V. Nucleic acid for a1at regulation

Family Cites Families (23)

* 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
US5871974A (en) * 1990-09-28 1999-02-16 Ixsys Inc. Surface expression libraries of heteromeric receptors
NZ308772A (en) 1995-05-17 1999-04-29 Du Pont Recombinant baculovirus insecticides
FR2774699B1 (fr) 1997-11-17 2003-10-03 Rhone Poulenc Rorer Sa Methode de reduction des evenements de recombinaison homologue
WO2000016888A1 (en) 1998-09-24 2000-03-30 Ultrasonus Ab A mixing method and a mixing chamber
US6103526A (en) 1998-10-08 2000-08-15 Protein Sciences Corporation Spodoptera frugiperda single cell suspension cell line in serum-free media, methods of producing and using
DE60039880D1 (en) 1999-06-24 2008-09-25 Amsterdam Molecular Therapeutics Bv Therapie mit lipoproteinlipase (lpl) variant
EP1212411A2 (en) * 1999-08-20 2002-06-12 The Johns Hopkins University School Of Medicine Methods and compositions for the construction and use of fusion libraries
JP2003526377A (ja) * 2000-03-14 2003-09-09 ニユーロロジクス・インコーポレーテツド キメラキャプシドベクターの製造
US20030129203A1 (en) * 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
WO2003042361A2 (en) 2001-11-09 2003-05-22 Government Of The United States Of America, Department Of Health And Human Services Production of adeno-associated virus in insect cells
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
CA2369985A1 (en) * 2002-01-18 2003-07-18 Duke University Generation of recombinant adeno-associated viral vectors by a complete adenovirus-mediated approach
WO2003068993A1 (en) * 2002-02-14 2003-08-21 Novavax, Inc. Method for isolation and purification of expressed gene products in vitro
WO2003074714A1 (en) 2002-03-05 2003-09-12 Stichting Voor De Technische Wetenschappen Baculovirus expression system
DE10260805A1 (de) 2002-12-23 2004-07-22 Geneart Gmbh Verfahren und Vorrichtung zum Optimieren einer Nucleotidsequenz zur Expression eines Proteins
AU2003272868A1 (en) * 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
DK1776460T3 (da) 2004-08-03 2010-04-12 Geneart Ag Fremgangsmåde til modulering af genekspression ved ændring af CpG-indholdet
EP1945779B1 (en) * 2005-10-20 2013-03-06 UniQure IP B.V. Improved aav vectors produced in insect cells
US20090191597A1 (en) * 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
CN103849629B (zh) * 2006-06-21 2017-06-09 尤尼克尔Ip股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav‑rep78翻译起始密码子的载体
SI2173888T1 (sl) * 2007-07-26 2017-01-31 Uniqure Ip B.V. Bakulovirusni vektorji, ki vsebujejo ponavljajoča kodirna zaporedja z različnimi kodonskimi preferencami

Similar Documents

Publication Publication Date Title
JP2014239686A5 (enExample)
US20240093231A1 (en) Aav capsid production in insect cells
US20240344085A1 (en) Altering tissue tropism of adeno-associated viruses
JP2020062045A (ja) 昆虫細胞で産生される、さらに改善されたaavベクター
IL317938A (en) Recombinant viral vectors with altered tropism and their uses for the targeted introduction of genetic material into human cells
FI3684423T3 (fi) Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
TW202005978A (zh) 新穎肝靶向腺相關病毒載體
JP2017536116A5 (enExample)
KR20190032375A (ko) 신규 아데노-관련 바이러스 캡시드 단백질
FI3313991T3 (fi) Muunnettu tekijä IX sekä koostumukset, menetelmät ja käytöt geeninsiirtoa varten soluihin, elimiin ja kudoksiin
JP2012519008A5 (enExample)
JP2014012022A5 (enExample)
JP2010538675A (ja) タンパク質産生の改善のためのaav複製機構の使用
JPWO2020223231A5 (enExample)
JPWO2020223236A5 (enExample)
TW202134440A (zh) 用於生產重組aav之新穎組合物及方法
CN111718418A (zh) 一种增强基因编辑的融合蛋白及其应用
WO2023225545A3 (en) Liver de-targeted recombinant aav capsid proteins
JP2009077737A5 (enExample)
AR123956A1 (es) Cápsides de aav y composiciones que las contienen
RU2021133906A (ru) Мутант aav, обладающий способностью нацеливаться на головной мозг
CN121271808A (zh) 一种生产低空壳率腺相关病毒的重组杆状病毒及其应用
HK40044351B (en) Liver-specific tropism of adeno-associated viruses
HK40044351A (en) Liver-specific tropism of adeno-associated viruses