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Gene therapies for mucopolysaccharidoses

Journal of inherited metabolic disease, 2024-01, Vol.47 (1), p.135-144

2024

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Liver‐directed gene therapy for inherited metabolic diseases

Journal of inherited metabolic disease, 2024-01, Vol.47 (1), p.9-21

2024

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Ammonia and autophagy: An emerging relationship with implications for disorders with hyperammonemia

Journal of inherited metabolic disease, 2019-11, Vol.42 (6), p.1097-1104

2019

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New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency

Journal of inherited metabolic disease, 2023-03, Vol.46 (2), p.335-347

2023

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Liver‐directed gene therapy for ornithine aminotransferase deficiency

EMBO molecular medicine, 2023-04, Vol.15 (4), p.e17033-n/a

2023

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Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha‐1‐anti‐trypsin deficiency

EMBO molecular medicine, 2013-03, Vol.5 (3), p.397-412

2013

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Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders

EMBO molecular medicine, 2021-02, Vol.13 (2), p.e13158-n/a

2021

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Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency

EMBO molecular medicine, 2023-03, Vol.15 (3), p.e14837-n/a

2023

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Hepatic glutamine synthetase augmentation enhances ammonia detoxification

Journal of inherited metabolic disease, 2019-11, Vol.42 (6), p.1128-1135

2019

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Liver gene therapy: The magic bullet for the sick lung

Molecular therapy. Methods & clinical development, 2022-09, Vol.26, p.72-73

Brunetti-Pierri, Nicola

2022

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A retrograde approach for liver gene transfer

Molecular therapy. Methods & clinical development, 2022-12, Vol.27, p.488-490

2022

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Differential inhibition of PDKs by phenylbutyrate and enhancement of pyruvate dehydrogenase complex activity by combination with dichloroacetate

Journal of inherited metabolic disease, 2015-09, Vol.38 (5), p.895-904

2015

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A gene silencing-based approach to tackle fatty liver disease

Molecular therapy. Methods & clinical development, 2024-03, Vol.32 (1), p.101198-101198, Article 101198

2024

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Efficient, Long-term Hepatic Gene Transfer Using Clinically Relevant HDAd Doses by Balloon Occlusion Catheter Delivery in Nonhuman Primates

Molecular therapy, 2009-02, Vol.17 (2), p.327-333

2009

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Challenges and Prospects for Helper-Dependent Adenoviral Vector-Mediated Gene Therapy

Biomedicines, 2014-04, Vol.2 (2), p.132-148

2014

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Intrathecal injection of helper-dependent adenoviral vectors results in long-term transgene expression in neuroependymal cells and neurons

Human gene therapy, 2011-06, Vol.22 (6), p.745-751

2011

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Vision on gyrate atrophy: why treat the liver?

EMBO molecular medicine, 2024-01, Vol.16 (1), p.8-9

2024

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Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1

Gene therapy, 2016-02, Vol.23 (2), p.129-134

2016

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SR-A and SREC-I binding peptides increase HDAd-mediated liver transduction

Gene therapy, 2014-11, Vol.21 (11), p.950-957

2014

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Retinal transduction profiles by high-capacity viral vectors

Gene therapy, 2014-10, Vol.21 (10), p.855-865

2014

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A systematic cross-sectional survey of multiple sulfatase deficiency

Molecular genetics and metabolism, 2020-08, Vol.130 (4), p.283-288

2020

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Liver-directed gene-based therapies for inborn errors of metabolism

Expert opinion on biological therapy, 2021-02, Vol.21 (2), p.229-240

2021

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Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism

Human gene therapy, 2019-10, Vol.30 (10), p.1204-1210

2019

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Gene therapy for inherited diseases of liver metabolism

Human gene therapy, 2015-04, Vol.26 (4), p.186-192

2015

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Two familial cases of high blood galactose of unknown aetiology

Journal of inherited metabolic disease, 2006-12, Vol.29 (6), p.762-762

2006

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Gene therapies for mucopolysaccharidoses

Liver‐directed gene therapy for inherited metabolic diseases

Ammonia and autophagy: An emerging relationship with implications for disorders with hyperammonemia

New mouse models with hypomorphic SUMF1 variants mimic attenuated forms of multiple sulfatase deficiency

Liver‐directed gene therapy for ornithine aminotransferase deficiency

Gene transfer of master autophagy regulator TFEB results in clearance of toxic protein and correction of hepatic disease in alpha‐1‐anti‐trypsin deficiency

Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders

Drug screening identifies tazarotene and bexarotene as therapeutic agents in multiple sulfatase deficiency

Hepatic glutamine synthetase augmentation enhances ammonia detoxification

Liver gene therapy: The magic bullet for the sick lung

A retrograde approach for liver gene transfer

Differential inhibition of PDKs by phenylbutyrate and enhancement of pyruvate dehydrogenase complex activity by combination with dichloroacetate

A gene silencing-based approach to tackle fatty liver disease

Efficient, Long-term Hepatic Gene Transfer Using Clinically Relevant HDAd Doses by Balloon Occlusion Catheter Delivery in Nonhuman Primates

Challenges and Prospects for Helper-Dependent Adenoviral Vector-Mediated Gene Therapy

Intrathecal injection of helper-dependent adenoviral vectors results in long-term transgene expression in neuroependymal cells and neurons

Vision on gyrate atrophy: why treat the liver?

Helper-dependent adenoviral vectors for liver-directed gene therapy of primary hyperoxaluria type 1

SR-A and SREC-I binding peptides increase HDAd-mediated liver transduction

Retinal transduction profiles by high-capacity viral vectors

A systematic cross-sectional survey of multiple sulfatase deficiency

Liver-directed gene-based therapies for inborn errors of metabolism

Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism

Gene therapy for inherited diseases of liver metabolism

Two familial cases of high blood galactose of unknown aetiology

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