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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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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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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

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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LRRK2 phosphorylates novel tau epitopes and promotes tauopathy

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

2013

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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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Highlighting hybridization: a case report of virtual reality-augmented interventions to improve chronic post-stroke recovery

Medicine (Baltimore), 2022-06, Vol.101 (25), p.e29357-e29357

Bailey, Rachel Blanchette

2022

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Human neutralizing antibodies against SARS-CoV-2 require intact Fc effector functions for optimal therapeutic protection

Cell, 2021-04, Vol.184 (7), p.1804-1820.e16

2021

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Awareness, Attitudes, and Actions Related to COVID-19 Among Adults With Chronic Conditions at the Onset of the U.S. Outbreak: A Cross-sectional Survey

Annals of internal medicine, 2020-07, Vol.173 (2), p.100

2020

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The HIF complex recruits the histone methyltransferase SET1B to activate specific hypoxia-inducible genes

Nature genetics, 2021-07, Vol.53 (7), p.1022-1035

2021

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Closed-Loop Therapy and Preservation of C-Peptide Secretion in Type 1 Diabetes

The New England journal of medicine, 2022-09, Vol.387 (10), p.882-893

2022

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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

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

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

LRRK2 phosphorylates novel tau epitopes and promotes tauopathy

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

Highlighting hybridization: a case report of virtual reality-augmented interventions to improve chronic post-stroke recovery

Human neutralizing antibodies against SARS-CoV-2 require intact Fc effector functions for optimal therapeutic protection

Awareness, Attitudes, and Actions Related to COVID-19 Among Adults With Chronic Conditions at the Onset of the U.S. Outbreak: A Cross-sectional Survey

Epithelial NOTCH Signaling Rewires the Tumor Microenvironment of Colorectal Cancer to Drive Poor-Prognosis Subtypes and Metastasis

The HIF complex recruits the histone methyltransferase SET1B to activate specific hypoxia-inducible genes

Closed-Loop Therapy and Preservation of C-Peptide Secretion in Type 1 Diabetes

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