Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.

Humans Dependovirus / genetics Liver / metabolism Genetic Therapy / methods Hepatocytes / metabolism Capsid Proteins / metabolism Tropism Genetic Vectors / genetics
AAV hepatocyte liver gene therapy nonhuman primate xenograft model

Journal

Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950

Informations de publication

Date de publication:
04 2023
Historique:
medline: 24 4 2023
pubmed: 18 3 2023
entrez: 17 3 2023
Statut: ppublish

Résumé

The liver is a prime target for

Identifiants

DOI: 10.1089/hum.2022.188 PMID: 36927149 PMC: PMC10150726
pubmed: 36927149
doi: 10.1089/hum.2022.188
pmc: PMC10150726
doi:

Substances chimiques

Capsid Proteins 0

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

273-288

Subventions

Organisme : Medical Research Council
ID : MR/T008024/1
Pays : United Kingdom
Organisme : Department of Health
Pays : United Kingdom
Organisme : Wellcome Trust
ID : 217112/Z/19/Z
Pays : United Kingdom

Commentaires et corrections

Type : ErratumIn

Références

J Virol. 1998 Jan;72(1):309-19
pubmed: 9420229
Front Med (Lausanne). 2022 Feb 09;8:809118
pubmed: 35223884
Mol Ther. 2020 Sep 2;28(9):2073-2082
pubmed: 32559433
Biomater Sci. 2022 Oct 11;10(20):5876-5887
pubmed: 36149407
Lancet. 2017 Aug 26;390(10097):849-860
pubmed: 28712537
Nat Nanotechnol. 2022 Mar;17(3):310-318
pubmed: 35132167
Mol Ther. 2005 Jun;11(6):875-88
pubmed: 15922958
Nat Med. 2007 Apr;13(4):419-22
pubmed: 17369837
Mol Ther Methods Clin Dev. 2020 May 13;17:1139-1154
pubmed: 32490035
iScience. 2020 Sep 28;23(10):101621
pubmed: 33089109
Biomed Pharmacother. 2019 Feb;110:493-499
pubmed: 30530229
Mol Ther Methods Clin Dev. 2018 Nov 01;12:71-84
pubmed: 30534580
Mol Ther Methods Clin Dev. 2019 May 28;14:27-36
pubmed: 31276009
Blood. 2002 Apr 15;99(8):2670-6
pubmed: 11929752
Mol Ther. 2015 Dec;23(12):1877-87
pubmed: 26412589
Proc Natl Acad Sci U S A. 1966 Jun;55(6):1467-74
pubmed: 5227666
Nat Commun. 2022 Oct 10;13(1):5947
pubmed: 36210364
Hum Gene Ther. 2020 May;31(9-10):575-589
pubmed: 32000541
Hum Pathol. 2009 Oct;40(10):1512-5
pubmed: 19751877
JHEP Rep. 2021 Apr 30;3(4):100300
pubmed: 34159305
EMBO J. 1988 Jul;7(7):2075-87
pubmed: 3262058
Hum Gene Ther. 2010 Jun;21(6):704-12
pubmed: 20095819
Mol Ther Methods Clin Dev. 2021 Apr 24;21:607-620
pubmed: 34095344
Hum Gene Ther Methods. 2013 Apr;24(2):59-67
pubmed: 23442094
J Inherit Metab Dis. 2017 Jul;40(4):497-517
pubmed: 28567541
Annu Rev Virol. 2014 Nov;1(1):427-51
pubmed: 26958729
Proc Natl Acad Sci U S A. 1982 Mar;79(6):2077-81
pubmed: 6281795
Nat Biotechnol. 2007 Aug;25(8):903-10
pubmed: 17664939
Proc Natl Acad Sci U S A. 2002 Sep 3;99(18):11854-9
pubmed: 12192090
Eur J Pharm Sci. 2021 Aug 1;163:105876
pubmed: 33989755
Science. 2018 Jan 12;359(6372):
pubmed: 29326244
N Engl J Med. 2017 Nov 2;377(18):1713-1722
pubmed: 29091557
Mol Ther Methods Clin Dev. 2020 Jan 21;16:192-203
pubmed: 32055647
Nature. 2014 Feb 20;506(7488):382-6
pubmed: 24390344
Front Immunol. 2021 Mar 17;12:658399
pubmed: 33815421
Hum Gene Ther Methods. 2015 Apr;26(2):45-53
pubmed: 25819687
Genomics. 1999 May 1;57(3):455-8
pubmed: 10329016
Mol Ther. 2020 Mar 4;28(3):723-746
pubmed: 31972133
Mol Ther. 2018 Jan 3;26(1):289-303
pubmed: 29055620
J Virol. 2006 Jan;80(1):426-39
pubmed: 16352567
Sci Rep. 2020 Jan 21;10(1):864
pubmed: 31965041
Nat Commun. 2020 Oct 28;11(1):5432
pubmed: 33116134
Gene Ther. 2012 Mar;19(3):288-94
pubmed: 21697954
N Engl J Med. 2021 Nov 18;385(21):1961-1973
pubmed: 34788507
Hum Gene Ther. 2019 Jan;30(1):79-87
pubmed: 30027761
Blood. 2018 Mar 1;131(9):1022-1031
pubmed: 29246900
Virology. 1984 Apr 15;134(1):52-63
pubmed: 6324476
J Integr Med. 2015 Sep;13(5):341-6
pubmed: 26343106
J Virol. 2004 Mar;78(6):3110-22
pubmed: 14990730
Nat Commun. 2014;5:3075
pubmed: 24435020
Hum Gene Ther. 2011 Nov;22(11):1389-401
pubmed: 21476868
Blood Adv. 2019 Nov 12;3(21):3241-3247
pubmed: 31698454
Methods Mol Biol. 2019;1576:123-133
pubmed: 27704362
Nat Med. 2020 Jul;26(7):1096-1101
pubmed: 32483358
Cancer Res. 1982 Sep;42(9):3858-63
pubmed: 6286115
N Engl J Med. 2011 Dec 22;365(25):2357-65
pubmed: 22149959
Gene Ther. 2013 Apr;20(4):361-9
pubmed: 22717743
N Engl J Med. 2014 Nov 20;371(21):1994-2004
pubmed: 25409372
Hum Gene Ther. 2022 Jun;33(11-12):664-682
pubmed: 35297686
J Virol. 1983 Feb;45(2):555-64
pubmed: 6300419
Mol Ther. 2006 Sep;14(3):316-27
pubmed: 16824801
Mol Ther Methods Clin Dev. 2021 Nov 25;24:88-101
pubmed: 34977275
Sci Transl Med. 2020 Sep 9;12(560):
pubmed: 32908003
JCI Insight. 2020 Sep 17;5(19):
pubmed: 32941184
Int J Biol Sci. 2019 Mar 1;15(4):847-856
pubmed: 30906215
Gene Ther. 2017 Jan;24(1):49-59
pubmed: 27834949
Mol Ther. 2010 Jan;18(1):126-34
pubmed: 19888196
J Biol Chem. 2019 Sep 20;294(38):14096-14104
pubmed: 31366731
Blood Adv. 2019 Sep 10;3(17):2632-2641
pubmed: 31501158
Nat Med. 2006 Mar;12(3):342-7
pubmed: 16474400
Blood. 2007 Feb 15;109(4):1414-21
pubmed: 17090654
Gut. 2019 Dec;68(12):2228-2237
pubmed: 31300517
J Biol Chem. 1993 Apr 15;268(11):8221-9
pubmed: 7681840
Clin Vaccine Immunol. 2011 Sep;18(9):1586-8
pubmed: 21775517

Auteurs

Adrian Westhaus (A)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
ORCID: 0000-0001-6439-4326

Marti Cabanes-Creus (M)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.

Kimberley L Dilworth (KL)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.

Erhua Zhu (E)

Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.

David Salas Gómez (D)

Gene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain.

Renina G Navarro (RG)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.

Anais K Amaya (AK)

Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
ORCID: 0000-0002-8396-2226

Suzanne Scott (S)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation, Sydney, Australia.

Magdalena Kwiatek (M)

Military Institute of Hygiene and Epidemiology, The Biological Threats Identification and Countermeasure Centre, Puławy, Poland.

Alexandra L McCorkindale (AL)

Inventia Life Science Pty Ltd., Sydney, Australia.

Tara E Hayman (TE)

Inventia Life Science Pty Ltd., Sydney, Australia.

Silke Frahm (S)

Stem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany.

Dany P Perocheau (DP)

Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.
National Institute of Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom.

Bang Manh Tran (BM)

Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.

Elizabeth Vincan (E)

Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.
Victorian Infectious Diseases Reference Laboratory, Royal Melbourne Hospital at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.
Curtin Medical School, Curtin University, Perth, Australia.

Sharon L Wong (SL)

Molecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia.
School of Biomedical Sciences, Faculty of Medicine, University of New South Wales, Sydney, Australia.

Shafagh A Waters (SA)

Molecular and Integrative Cystic Fibrosis (miCF) Research Centre, University of New South Wales and Sydney Children's Hospital, Sydney, Australia.
School of Biomedical Sciences, Faculty of Medicine, University of New South Wales, Sydney, Australia.
Department of Respiratory Medicine, Sydney Children's Hospital, Faculty of Medicine, University of New South Wales, Sydney, Australia.

Georgina E Riddiough (GE)

Department of Infectious Diseases, Melbourne Medical School, The University of Melbourne at the Peter Doherty Institute for Infection and Immunity, Melbourne, Australia.
Department of Surgery, Austin Health Precinct, The University of Melbourne, Austin Health, Heidelberg, Australia.

Marcos V Perini (MV)

Department of Surgery, Austin Health Precinct, The University of Melbourne, Austin Health, Heidelberg, Australia.

Laurence O W Wilson (LOW)

Australian e-Health Research Centre, Commonwealth Scientific and Industrial Research Organisation, Sydney, Australia.
Applied BioSciences, Faculty of Science and Engineering, Macquarie University, Sydney, Australia.

Julien Baruteau (J)

Genetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
Metabolic Medicine Department, Great Ormond Street Hospital for Children NHS Foundation Trust, London, United Kingdom.
National Institute of Health Research Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom.

Sebastian Diecke (S)

Stem Cell Technology Platform, Max Delbrück Centrum for Molecular Medicine, Berlin, Germany.

Gloria González-Aseguinolaza (G)

Gene Therapy and Regulation of Gene Expression Department, IdiSNA, Instituto de Investigación Sanitaria de Navarra, Universidad de Navarra, CIMA, Pamplona, Spain.
ORCID: 0000-0002-1600-4562

Giorgia Santilli (G)

Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
ORCID: 0000-0003-1776-1984

Adrian J Thrasher (AJ)

Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.
ORCID: 0000-0002-6097-6115

Ian E Alexander (IE)

Gene Therapy Research Unit, Children's Medical Research Institute and Sydney Children's Hospitals Network, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Discipline of Child and Adolescent Health, Faculty of Medicine and Health, The University of Sydney, Sydney, Australia.

Leszek Lisowski (L)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Vector and Genome Engineering Facility, Children's Medical Research Institute, Faculty of Medicine and Health, The University of Sydney, Westmead, Australia.
Laboratory of Molecular Oncology and Innovative Therapies, Military Institute of Medicine, Warszawa, Poland.
ORCID: 0000-0002-4772-8651

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female

Evaluation of Low-Value Services Across Major Medicare Advantage Insurers and Traditional Medicare.

Ciara Duggan, Adam L Beckman, Ishani Ganguli et al.
1.00
Humans United States Aged Cross-Sectional Studies Medicare Part C

Effectiveness of Virtual Yoga for Chronic Low Back Pain: A Randomized Clinical Trial.

Hallie Tankha, Devyn Gaskins, Amanda Shallcross et al.
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Virus › Virus à ADN › Parvoviridae › Parvovirinae › Dependovirus › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Système digestif › Foie › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Cellules › Cellules épithéliales › Hépatocytes › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Acides aminés, peptides et protéines › Protéines › Protéines virales › Protéines virales structurales › Protéines nucléocapside › Protéines de capside › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Phénomènes physiologiques des plantes › Tropisme › Assessment of Pre-Clinical Liver Models Based...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Vecteurs génétiques › Assessment of Pre-Clinical Liver Models Based...