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Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies

Brain research, 2020-07, Vol.1739, p.146832-146832, Article 146832

2020

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Lrrk promotes tau neurotoxicity through dysregulation of actin and mitochondrial dynamics

PLoS biology, 2018-12, Vol.16 (12), p.e2006265

2018

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Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.160-171

2018

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Editorial: Personalized medicine for neuromuscular disorders

Frontiers in cell and developmental biology, 2023-11, Vol.11, p.1329048-1329048

2023

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Akt and CHIP Coregulate Tau Degradation through Coordinated Interactions

Proceedings of the National Academy of Sciences - PNAS, 2008-03, Vol.105 (9), p.3622-3627

2008

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SLC13A5 Deficiency Disorder: From Genetics to Gene Therapy

Genes, 2022-09, Vol.13 (9), p.1655

2022

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Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies

Frontiers in neurology, 2022-06, Vol.13, p.805007-805007

2022

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Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens

Orphanet journal of rare diseases, 2019-02, Vol.14 (1), p.27-27, Article 27

2019

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Organic anion transporter 3 (Oat3/Slc22a8) knockout mice exhibit altered clearance and distribution of penicillin G

American journal of physiology. Renal physiology, 2007-10, Vol.293 (4), p.F1332-F1341

2007

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In vivo functional brain mapping in a conditional mouse model of human tauopathy (tauP301L) reveals reduced neural activity in memory formation structures

Molecular neurodegeneration, 2013-02, Vol.8 (1), p.9-9, Article 9

2013

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Effects of the C57BL/6 strain background on tauopathy progression in the rTg4510 mouse model

Molecular neurodegeneration, 2014-01, Vol.9 (1), p.8-8, Article 8

2014

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Gene Transfer Therapy for Neurodevelopmental Disorders

Developmental neuroscience, 2021-09, Vol.43 (3-4), p.230-240

2021

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AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

The Journal of clinical investigation, 2021-06, Vol.131 (11), p.1-11

2021

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Identification of potential protein interactors of Lrrk2

Parkinsonism & related disorders, 2007-10, Vol.13 (7), p.382-385

2007

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The prolyl hydroxylase oxygen-sensing pathway is cytoprotective and allows maintenance of mitochondrial membrane potential during metabolic inhibition

American Journal of Physiology: Cell Physiology, 2007-02, Vol.292 (2), p.C719-C728

2007

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Intrathecal Gene Therapy for Giant Axonal Neuropathy

The New England journal of medicine, 2024-03, Vol.390 (12), p.1092-1104

2024

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Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease

Ophthalmic genetics, 2021-10, Vol.42 (5), p.600-603

2021

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Evaluating Learning Outcomes and Assessing Social Work Skill Development: Comparing Online vs. In-Person Education

Journal of technology in human services, 2022-01, Vol.40 (1), p.47-57

2022

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Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease

Clinical autonomic research, 2016-08, Vol.26 (4), p.307-313

2016

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Erratum to: Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease

Clinical autonomic research, 2016-12, Vol.26 (6), p.467-467

2016

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Efficacy of a scAAV9/SUMF1 viral vector for the treatment of multiple sulfatase deficiency

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S101-S101

2022

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Efficacy of a scAAV9/ viral vector for the treatment of multiple sulfatase deficiency

Molecular genetics and metabolism, 2022-02, Vol.135 (2), p.S101

2022

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Development of scAAV9/SUMF1 gene therapy for multiple sulfatase deficiency

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S27-S27

2019

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Preclinical studies to support the intrathecal delivery of scAAV9/SUMF1 as a gene replacement therapy for multiple sulfatase deficiency

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S17-S18

2021

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604. Comparison of Intra-Cisterna Magna and Lumbar Puncture Intrathecal Delivery of scAAV9 GeneTherapy for Giant Axonal Neuropathy

Molecular therapy, 2016-05, Vol.24, p.S239-S239

2016

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Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies

Lrrk promotes tau neurotoxicity through dysregulation of actin and mitochondrial dynamics

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

Editorial: Personalized medicine for neuromuscular disorders

Akt and CHIP Coregulate Tau Degradation through Coordinated Interactions

SLC13A5 Deficiency Disorder: From Genetics to Gene Therapy

Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies

Advancing the pathologic phenotype of giant axonal neuropathy: early involvement of the ocular lens

Organic anion transporter 3 (Oat3/Slc22a8) knockout mice exhibit altered clearance and distribution of penicillin G

In vivo functional brain mapping in a conditional mouse model of human tauopathy (tauP301L) reveals reduced neural activity in memory formation structures

Effects of the C57BL/6 strain background on tauopathy progression in the rTg4510 mouse model

Gene Transfer Therapy for Neurodevelopmental Disorders

AAV9-mediated FIG4 delivery prolongs life span in Charcot-Marie-Tooth disease type 4J mouse model

Identification of potential protein interactors of Lrrk2

The prolyl hydroxylase oxygen-sensing pathway is cytoprotective and allows maintenance of mitochondrial membrane potential during metabolic inhibition

Intrathecal Gene Therapy for Giant Axonal Neuropathy

Extensive rod and cone photoreceptor-cell degeneration in rat models of giant axonal neuropathy: implications for gene therapy of human disease

Evaluating Learning Outcomes and Assessing Social Work Skill Development: Comparing Online vs. In-Person Education

Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease

Erratum to: Autonomic nervous system involvement in the giant axonal neuropathy (GAN) KO mouse: implications for human disease

Efficacy of a scAAV9/SUMF1 viral vector for the treatment of multiple sulfatase deficiency

Efficacy of a scAAV9/ viral vector for the treatment of multiple sulfatase deficiency

Development of scAAV9/SUMF1 gene therapy for multiple sulfatase deficiency

Preclinical studies to support the intrathecal delivery of scAAV9/SUMF1 as a gene replacement therapy for multiple sulfatase deficiency

604. Comparison of Intra-Cisterna Magna and Lumbar Puncture Intrathecal Delivery of scAAV9 GeneTherapy for Giant Axonal Neuropathy

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