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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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Toward a translational gene therapy for mucolipidosis IV

Molecular therapy. Methods & clinical development, 2024-12, Vol.32 (4), p.101345, Article 101345

2024

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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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The high-affinity HSP90-CHIP complex recognizes and selectively degrades phosphorylated tau client proteins

The Journal of clinical investigation, 2007-03, Vol.117 (3), p.648-658

2007

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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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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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Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice

Gene therapy, 2024-11

2024

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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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LRRK2 phosphorylates novel tau epitopes and promotes tauopathy

Acta neuropathologica, 2013-12, Vol.126 (6), p.809-827

2013

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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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Sleep Abnormalities in SLC13A5 Citrate Transporter Disorder

Genes, 2024-10, Vol.15 (10), p.1338

2024

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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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SLC13A5 Deficiency Disorder: From Genetics to Gene Therapy

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

2022

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Methods for Gene Transfer to the Central Nervous System

Advances in Genetics, 2014, Vol.87, p.125-197

2014

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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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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

Toward a translational gene therapy for mucolipidosis IV

Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

The high-affinity HSP90-CHIP complex recognizes and selectively degrades phosphorylated tau client proteins

Editorial: Personalized medicine for neuromuscular disorders

Akt and CHIP Coregulate Tau Degradation through Coordinated Interactions

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

Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice

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

LRRK2 phosphorylates novel tau epitopes and promotes tauopathy

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

Sleep Abnormalities in SLC13A5 Citrate Transporter Disorder

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

SLC13A5 Deficiency Disorder: From Genetics to Gene Therapy

Methods for Gene Transfer to the Central Nervous System

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

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