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AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/−SMA model mice

Human molecular genetics, 2019-10, Vol.28 (19), p.3199-3210

2019

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Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases

PLoS genetics, 2017-03, Vol.13 (3), p.e1006680-e1006680

2017

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A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.348-360

2018

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The adeno-associated virus 2 genome and Rep 68/78 proteins interact with cellular sites of DNA damage

Human molecular genetics, 2022-03, Vol.31 (6), p.985-998

2022

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AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice

Biochemical and biophysical research communications, 2020-09, Vol.530 (1), p.107-114

2020

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Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy

Molecular therapy, 2016-09, Vol.24 (9), p.1592-1601

2016

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Functional characterization of SMN evolution in mouse models of SMA

Scientific reports, 2019-07, Vol.9 (1), p.9472-12, Article 9472

2019

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Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCFSlmb degron

Molecular biology of the cell, 2018-01, Vol.29 (2), p.96-110

2018

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Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy

JCI insight, 2017-03, Vol.2 (5), p.e89970-e89970

2017

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Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCF Slmb degron

Molecular biology of the cell, 2018-01, Vol.29 (2), p.96-110

2018

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AAV9-DOK7 gene therapy reduces disease severity in Smn SMA model mice

Biochemical and biophysical research communications, 2020-09, Vol.530 (1), p.107-114

2020

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AAV9-DOK7 gene therapy reduces disease severity in Smn 2B/- SMA model mice

Biochemical and biophysical research communications, 2020-09, Vol.530 (1), p.107

2020

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4PD Functionalized Dendrimers: A Flexible Tool for In Vivo Gene Silencing of Tumor-Educated Myeloid Cells

The Journal of immunology (1950), 2017-05, Vol.198 (10), p.4166-4177

2017

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AAV9-mediated delivery of miR-23a reduces disease severity in Smn2B/−SMA model mice

Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases

A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1

The adeno-associated virus 2 genome and Rep 68/78 proteins interact with cellular sites of DNA damage

AAV9-DOK7 gene therapy reduces disease severity in Smn2B/- SMA model mice

Optimization of Morpholino Antisense Oligonucleotides Targeting the Intronic Repressor Element1 in Spinal Muscular Atrophy

Functional characterization of SMN evolution in mouse models of SMA

Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCFSlmb degron

Plastin-3 extends survival and reduces severity in mouse models of spinal muscular atrophy

Self-oligomerization regulates stability of survival motor neuron protein isoforms by sequestering an SCF Slmb degron

AAV9-DOK7 gene therapy reduces disease severity in Smn SMA model mice

AAV9-DOK7 gene therapy reduces disease severity in Smn 2B/- SMA model mice

4PD Functionalized Dendrimers: A Flexible Tool for In Vivo Gene Silencing of Tumor-Educated Myeloid Cells

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