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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

Nature communications, 2021-11, Vol.12 (1), p.6769-12, Article 6769

2021

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Evaluation of muscle function of the extensor digitorum longus muscle ex vivo and tibialis anterior muscle in situ in mice

Journal of visualized experiments, 2013-02 (72)

2013

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A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

Molecular therapy. Methods & clinical development, 2017-09, Vol.6 (C), p.216-230

2017

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The evolution of heart gene delivery vectors

The journal of gene medicine, 2011-10, Vol.13 (10), p.557-565

2011

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The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Human gene therapy, 2023-05, Vol.34 (9-10), p.459

2023

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Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Molecular therapy, 2020-03, Vol.28 (3), p.845-854

2020

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Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model

Human gene therapy, 2023-05, Vol.34 (9-10), p.449

2023

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Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine

Expert opinion on drug discovery, 2020-04, Vol.15 (4), p.443-456

2020

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100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16045-16045, Article 16045

2016

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Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy

Journal of molecular and cellular cardiology, 2017-01, Vol.102, p.45-52

2017

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AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

Molecular therapy, 2019-09, Vol.27 (9), p.1568-1585

2019

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Partial restoration of cardiac function with ΔPDZ nNOS in aged mdx model of Duchenne cardiomyopathy

Human molecular genetics, 2014-06, Vol.23 (12), p.3189-3199

2014

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The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy

Human gene therapy, 2022-05, Vol.33 (9-10), p.518

2022

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Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy

Human molecular genetics, 2013-07, Vol.22 (13), p.2634-2641

2013

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AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in > 21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy

Journal of molecular and cellular cardiology, 2012-08, Vol.53 (2), p.217-222

2012

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Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy

Human gene therapy, 2018-07, Vol.29 (7), p.737

2018

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Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype

Human molecular genetics, 2016-07, Vol.25 (13), p.2633-2644

2016

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Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin

Human molecular genetics, 2021-06, Vol.30 (14), p.1321-1336

2021

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Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy

Human gene therapy, 2019-05, Vol.30 (5), p.535

2019

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AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice

JCI insight, 2018-12, Vol.3 (23)

2018

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AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice

JCI insight, 2018-12, Vol.3 (23)

2018

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Dermacentor variabilis: Characterization and modeling of macrophage migration inhibitory factor with phylogenetic comparisons to other ticks, insects and parasitic nematodes

Experimental parasitology, 2012-03, Vol.130 (3), p.232-238

2012

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The FVB Background Does Not Dramatically Alter the Dystrophic Phenotype of Mdx Mice

PLoS currents, 2015-02, Vol.7

2015

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A white-tailed deer/lone star tick model for studying transmission of Ehrlichia chaffeensis

Vector borne and zoonotic diseases (Larchmont, N.Y.), 2013-03, Vol.13 (3), p.193

2013

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Expression and regulation of macrophage migration inhibitory factor (MIF) in feeding American dog ticks, Dermacentor variabilis

Experimental & applied acarology, 2012-06, Vol.57 (2), p.179-187

2012

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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

Evaluation of muscle function of the extensor digitorum longus muscle ex vivo and tibialis anterior muscle in situ in mice

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

The evolution of heart gene delivery vectors

The Implication of Hinge 1 and Hinge 4 in Micro-Dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy

Lifelong Outcomes of Systemic Adeno-Associated Virus Micro-Dystrophin Gene Therapy in a Murine Duchenne Muscular Dystrophy Model

Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine

100-fold but not 50-fold dystrophin overexpression aggravates electrocardiographic defects in the mdx model of Duchenne muscular dystrophy

Uniform low-level dystrophin expression in the heart partially preserved cardiac function in an aged mouse model of Duchenne cardiomyopathy

AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice

Partial restoration of cardiac function with ΔPDZ nNOS in aged mdx model of Duchenne cardiomyopathy

The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy

Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy

AAV micro-dystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in > 21-m-old mdx mice, an end-stage model of Duchenne muscular dystrophy cardiomyopathy

Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy

Genomic removal of a therapeutic mini-dystrophin gene from adult mice elicits a Duchenne muscular dystrophy-like phenotype

Proteomic analysis identifies key differences in the cardiac interactomes of dystrophin and micro-dystrophin

Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy

AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice

AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice

Dermacentor variabilis: Characterization and modeling of macrophage migration inhibitory factor with phylogenetic comparisons to other ticks, insects and parasitic nematodes

The FVB Background Does Not Dramatically Alter the Dystrophic Phenotype of Mdx Mice

A white-tailed deer/lone star tick model for studying transmission of Ehrlichia chaffeensis

Expression and regulation of macrophage migration inhibitory factor (MIF) in feeding American dog ticks, Dermacentor variabilis

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