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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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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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Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies

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

2022

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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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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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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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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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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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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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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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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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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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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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Preclinical gene therapy in a mouse model of Charcot-Marie-Tooth disease type 4J

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S96-S97

2019

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15. Development of Intrathecal scAAV9 Gene Therapy for Giant Axonal Neuropathy

Molecular therapy, 2015-05, Vol.23, p.S6-S7

2015

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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, Vol.62 (4)

2007

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Modulators of tauopathy in the rTg4510 mouse model

Bailey, Rachel M

2013

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

American Journal of Physiology: Cell Physiology, 2007, Vol.61 (2)

2007

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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, Vol.40 (1), p.47-57

2022

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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, Vol.42 (5), p.600-603

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

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

Gene Therapy: Novel Approaches to Targeting Monogenic Epilepsies

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

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

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

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

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

Toward a translational gene therapy for mucolipidosis IV

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

Efficacy of a scAAV9/SUMF1 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

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

Preclinical gene therapy in a mouse model of Charcot-Marie-Tooth disease type 4J

15. Development of Intrathecal scAAV9 Gene Therapy for Giant Axonal Neuropathy

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

Modulators of tauopathy in the rTg4510 mouse model

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

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

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

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

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