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Gene therapy clinical trials worldwide to 2017: An update

The journal of gene medicine, 2018-05, Vol.20 (5), p.e3015-n/a

2018

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

Nature (London), 2014-02, Vol.506 (7488), p.382-386

2014

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The implementation of newborn screening for spinal muscular atrophy: the Australian experience

Genetics in medicine, 2020-03, Vol.22 (3), p.557-565

2020

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Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Journal of inherited metabolic disease, 2017-07, Vol.40 (4), p.497-517

2017

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The Transcriptional and Functional Properties of Mouse Epiblast Stem Cells Resemble the Anterior Primitive Streak

Cell stem cell, 2014-01, Vol.14 (1), p.107-120

2014

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Great expectations: virus-mediated gene therapy in neurological disorders

Journal of neurology, neurosurgery and psychiatry, 2020-08, Vol.91 (8), p.849-860

2020

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Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV‐piggyBac Gene Therapy

Hepatology (Baltimore, Md.), 2019-12, Vol.70 (6), p.2047-2061

2019

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Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome

Nature genetics, 2017-08, Vol.49 (8), p.1267-1273

2017

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Lymphoid Regeneration from Gene-Corrected SCID-X1 Subject-Derived iPSCs

Cell stem cell, 2015-04, Vol.16 (4), p.367-372

2015

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Antigen expression level threshold tunes the fate of CD8 T cells during primary hepatic immune responses

Proceedings of the National Academy of Sciences - PNAS, 2014-06, Vol.111 (25), p.E2540-E2549

2014

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Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

Nature communications, 2024-03, Vol.15 (1), p.1876-1876, Article 1876

2024

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Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease

Journal of neuroinflammation, 2024-03, Vol.21 (1), p.77-77, Article 77

2024

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ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice

Molecular therapy, 2015-09, Vol.23 (9), p.1434-1443

2015

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Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

Annals of clinical and translational neurology, 2022-03, Vol.9 (3), p.339-350

2022

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Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Molecular therapy, 2008-06, Vol.16 (6), p.1081-1088

2008

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Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method

Viruses, 2022-07, Vol.14 (8), p.1623

2022

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Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.135-146

2021

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AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia

Molecular therapy. Methods & clinical development, 2024-06, Vol.32 (2), p.101232-101232, Article 101232

2024

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Ub-ISAP: a streamlined UNIX pipeline for mining unique viral vector integration sites from next generation sequencing data

BMC bioinformatics, 2017-06, Vol.18 (1), p.305-9, Article 305

2017

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Liver‐Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug‐Resistant Gene 2‐Knockout Mice

Hepatology communications, 2019-12, Vol.3 (12), p.1656-1673

2019

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Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system

Hepatology (Baltimore, Md.), 2015-08, Vol.62 (2), p.417-428

2015

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Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence

Nucleic acids research, 2014-09, Vol.42 (16), p.e129-e129

2014

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Gene therapy for urea cycle defects: An update from historical perspectives to future prospects

Journal of inherited metabolic disease, 2024-01, Vol.47 (1), p.50-62

2024

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Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

Molecular therapy. Methods & clinical development, 2022-03, Vol.24, p.88-101

2022

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Germline viral "fossils" guide in silico reconstruction of a mid-Cenozoic era marsupial adeno-associated virus

Scientific reports, 2016-07, Vol.6 (1), p.28965-28965, Article 28965

2016

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Gene therapy clinical trials worldwide to 2017: An update

Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

The implementation of newborn screening for spinal muscular atrophy: the Australian experience

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

The Transcriptional and Functional Properties of Mouse Epiblast Stem Cells Resemble the Anterior Primitive Streak

Great expectations: virus-mediated gene therapy in neurological disorders

Prevention of Cholestatic Liver Disease and Reduced Tumorigenicity in a Murine Model of PFIC Type 3 Using Hybrid AAV‐piggyBac Gene Therapy

Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome

Lymphoid Regeneration from Gene-Corrected SCID-X1 Subject-Derived iPSCs

Antigen expression level threshold tunes the fate of CD8 T cells during primary hepatic immune responses

Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation

Liver-specific adiponectin gene therapy suppresses microglial NLRP3-inflammasome activation for treating Alzheimer's disease

ACE2 Therapy Using Adeno-associated Viral Vector Inhibits Liver Fibrosis in Mice

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

Performance of Cardiotropic rAAV Vectors Is Dependent on Production Method

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

AAV-delivered hepato-adrenal cooperativity in steroidogenesis: Implications for gene therapy for congenital adrenal hyperplasia

Ub-ISAP: a streamlined UNIX pipeline for mining unique viral vector integration sites from next generation sequencing data

Liver‐Targeted Angiotensin Converting Enzyme 2 Therapy Inhibits Chronic Biliary Fibrosis in Multiple Drug‐Resistant Gene 2‐Knockout Mice

Modeling correction of severe urea cycle defects in the growing murine liver using a hybrid recombinant adeno‐associated virus/piggyBac transposase gene delivery system

Impact of next-generation sequencing error on analysis of barcoded plasmid libraries of known complexity and sequence

Gene therapy for urea cycle defects: An update from historical perspectives to future prospects

Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8

Germline viral "fossils" guide in silico reconstruction of a mid-Cenozoic era marsupial adeno-associated virus

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