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CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10

Molecular therapy, 2017-02, Vol.25 (2), p.331-341

2017

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DNA Damage-Induced Primordial Follicle Oocyte Apoptosis and Loss of Fertility Require TAp63-Mediated Induction of Puma and Noxa

Molecular cell, 2012-11, Vol.48 (3), p.343-352

2012

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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Neuron (Cambridge, Mass.), 2012-06, Vol.74 (6), p.1031-1044

2012

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Evaluation of inflammation and follicle depletion during ovarian ageing in mice

Scientific reports, 2021-01, Vol.11 (1), p.278-278, Article 278

2021

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The capacity of oocytes for DNA repair

Cellular and molecular life sciences : CMLS, 2018-08, Vol.75 (15), p.2777-2792

2018

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Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial

The Lancet (British edition), 2017-07, Vol.390 (10089), p.50-61

2017

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Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

Proceedings of the National Academy of Sciences - PNAS, 2017-03, Vol.114 (10), p.2699-2704

2017

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The importance of DNA repair for maintaining oocyte quality in response to anti-cancer treatments, environmental toxins and maternal ageing

Human reproduction update, 2018-03, Vol.24 (2), p.119-134

2018

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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Nature (London), 2012-08, Vol.488 (7409), p.111-115

2012

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Loss of PUMA protects the ovarian reserve during DNA-damaging chemotherapy and preserves fertility

Cell death & disease, 2018-05, Vol.9 (6), p.618-12, Article 618

2018

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Gene therapy for the neurological manifestations in lysosomal storage disorders

Journal of lipid research, 2014-09, Vol.55 (9), p.1827-1838

Cheng, Seng H.

2014

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Fetal gene therapy for neurodegenerative disease of infants

Nature medicine, 2018-09, Vol.24 (9), p.1317-1323

2018

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The Inflammasome Contributes to Depletion of the Ovarian Reserve During Aging in Mice

Frontiers in cell and developmental biology, 2021-02, Vol.8, p.628473-628473

2021

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Dimorphic effects of transforming growth factor-β signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome

Arteriosclerosis, thrombosis, and vascular biology, 2015-04, Vol.35 (4), p.911-917

2015

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Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease

Journal of neurochemistry, 2021-03, Vol.156 (5), p.692-701

2021

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Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

Science translational medicine, 2011-03, Vol.3 (72), p.72ra18

2011

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Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies

Proceedings of the National Academy of Sciences - PNAS, 2013-02, Vol.110 (9), p.3537-3542

2013

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Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis

Proceedings of the National Academy of Sciences - PNAS, 2015-06, Vol.112 (26), p.8100-8105

2015

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Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

Molecular therapy, 2023-03, Vol.31 (3), p.616-630

2023

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CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

Molecular therapy, 2016-06, Vol.24 (6), p.1019-1029

2016

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CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy

Proceedings of the National Academy of Sciences - PNAS, 2011-07, Vol.108 (29), p.12101-12106

2011

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Gaucher-related synucleinopathies: the examination of sporadic neurodegeneration from a rare (disease) angle

Progress in neurobiology, 2015-02, Vol.125, p.47-62

2015

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Yb-doped WO3 photocatalysts for water oxidation with visible light

International journal of hydrogen energy, 2014-03, Vol.39 (9), p.4291-4298

2014

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Effects of Biocompatible versus Standard Fluid on Peritoneal Dialysis Outcomes

Journal of the American Society of Nephrology, 2012-06, Vol.23 (6), p.1097-1107

2012

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Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

Science translational medicine, 2019-08, Vol.11 (506)

2019

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CRISPR/Cas9-Mediated Genome Editing as a Therapeutic Approach for Leber Congenital Amaurosis 10

DNA Damage-Induced Primordial Follicle Oocyte Apoptosis and Loss of Fertility Require TAp63-Mediated Induction of Puma and Noxa

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Evaluation of inflammation and follicle depletion during ovarian ageing in mice

The capacity of oocytes for DNA repair

Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial

Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models

The importance of DNA repair for maintaining oocyte quality in response to anti-cancer treatments, environmental toxins and maternal ageing

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Loss of PUMA protects the ovarian reserve during DNA-damaging chemotherapy and preserves fertility

Gene therapy for the neurological manifestations in lysosomal storage disorders

Fetal gene therapy for neurodegenerative disease of infants

The Inflammasome Contributes to Depletion of the Ovarian Reserve During Aging in Mice

Dimorphic effects of transforming growth factor-β signaling during aortic aneurysm progression in mice suggest a combinatorial therapy for Marfan syndrome

Substrate reduction therapy using Genz‐667161 reduces levels of pathogenic components in a mouse model of neuronopathic forms of Gaucher disease

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

Augmenting CNS glucocerebrosidase activity as a therapeutic strategy for parkinsonism and other Gaucher-related synucleinopathies

Glycosphingolipids are modulators of disease pathogenesis in amyotrophic lateral sclerosis

Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy

CNS-accessible Inhibitor of Glucosylceramide Synthase for Substrate Reduction Therapy of Neuronopathic Gaucher Disease

CNS expression of glucocerebrosidase corrects α-synuclein pathology and memory in a mouse model of Gaucher-related synucleinopathy

Gaucher-related synucleinopathies: the examination of sporadic neurodegeneration from a rare (disease) angle

Yb-doped WO3 photocatalysts for water oxidation with visible light

Effects of Biocompatible versus Standard Fluid on Peritoneal Dialysis Outcomes

Adeno-associated viral vector serotype 9–based gene therapy for Niemann-Pick disease type A

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