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Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

Annals of neurology, 2009-06, Vol.65 (6), p.667-676

2009

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Progressive Dystrophic Pathology in Diaphragm and Impairment of Cardiac Function in FKRP P448L Mutant Mice

PloS one, 2016-10, Vol.11 (10), p.e0164187-e0164187

2016

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Distinct expression of functionally glycosylated alpha-dystroglycan in muscle and non-muscle tissues of FKRP mutant mice

PloS one, 2018-01, Vol.13 (1), p.e0191016-e0191016

2018

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What Can We Learn From Clinical Trials of Exon Skipping for DMD?

Molecular therapy. Nucleic acids, 2014, Vol.3 (3), p.e152-e152, Article e152

2014

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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Nature medicine, 2006-02, Vol.12 (2), p.175-177

2006

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Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

Proceedings of the National Academy of Sciences - PNAS, 2008-09, Vol.105 (39), p.14814-14819

2008

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The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

Molecular therapy, 2011-01, Vol.19 (1), p.9-15

2011

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Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant mice

Nature communications, 2018-08, Vol.9 (1), p.3448-12, Article 3448

2018

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Ribitol alters multiple metabolic pathways of central carbon metabolism with enhanced glycolysis: A metabolomics and transcriptomics profiling of breast cancer

PloS one, 2022-12, Vol.17 (12), p.e0278711-e0278711

2022

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Ribitol alters multiple metabolic pathways of central carbon metabolism with enhanced glycolysis: A metabolomics and transcriptomics profiling of breast cancer

PloS one, 2022-12, Vol.17 (12)

2022

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Revertant Phenomenon in DMD and LGMD2I and Its Therapeutic Implications: A Review of Study Under Mentorship of Terrence Partridge

Journal of neuromuscular diseases, 2021, Vol.8 (s2), p.S359-S367

2021

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Skeletal, cardiac, and respiratory muscle function and histopathology in the P448Lneo- mouse model of FKRP-deficient muscular dystrophy

Skeletal muscle, 2018-04, Vol.8 (1), p.13-16, Article 13

2018

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Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model

PloS one, 2022-12, Vol.17 (12), p.e0278482-e0278482

2022

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Metabolomics Analysis of Skeletal Muscles from FKRP-Deficient Mice Indicates Improvement After Gene Replacement Therapy

Scientific reports, 2019-07, Vol.9 (1), p.10070-14, Article 10070

2019

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Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model

PloS one, 2022-12, Vol.17 (12)

2022

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Promiscuous expression of Epstein‐Barr virus genes in Burkitt's lymphoma from the central African country Malawi

International journal of cancer, 2002-06, Vol.99 (5), p.635-643

2002

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Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice

Molecular therapy, 2009-05, Vol.17 (5), p.864-871

2009

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In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice

Methods in molecular biology (Clifton, N.J.), 2018, Vol.1828, p.231

2018

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BCL-2 expression in adult and embryonic non-haematopoietic tissues

The Journal of pathology, 1993-04, Vol.169 (4), p.431-437

1993

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Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice

Molecular therapy. Methods & clinical development, 2018-12, Vol.11, p.106-120

2018

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A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

Cell reports (Cambridge), 2021-07, Vol.36 (2), p.109360-109360, Article 109360

2021

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ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant Mice

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.271-280

2020

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Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic mdx Mice by Peptide-Conjugated Morpholino

The American journal of pathology, 2012-08, Vol.181 (2), p.392-400

2012

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bcl-2: Role in epithelial differentiation and oncogenesis

Human pathology, 1996-02, Vol.27 (2), p.102-110

1996

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Systemic Delivery of Antisense Oligoribonucleotide Restores Dystrophin Expression in Body-Wide Skeletal Muscles

Proceedings of the National Academy of Sciences - PNAS, 2005-01, Vol.102 (1), p.198-203

2005

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Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

Progressive Dystrophic Pathology in Diaphragm and Impairment of Cardiac Function in FKRP P448L Mutant Mice

Distinct expression of functionally glycosylated alpha-dystroglycan in muscle and non-muscle tissues of FKRP mutant mice

What Can We Learn From Clinical Trials of Exon Skipping for DMD?

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy

Ribitol restores functionally glycosylated α-dystroglycan and improves muscle function in dystrophic FKRP-mutant mice

Ribitol alters multiple metabolic pathways of central carbon metabolism with enhanced glycolysis: A metabolomics and transcriptomics profiling of breast cancer

Ribitol alters multiple metabolic pathways of central carbon metabolism with enhanced glycolysis: A metabolomics and transcriptomics profiling of breast cancer

Revertant Phenomenon in DMD and LGMD2I and Its Therapeutic Implications: A Review of Study Under Mentorship of Terrence Partridge

Skeletal, cardiac, and respiratory muscle function and histopathology in the P448Lneo- mouse model of FKRP-deficient muscular dystrophy

Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model

Metabolomics Analysis of Skeletal Muscles from FKRP-Deficient Mice Indicates Improvement After Gene Replacement Therapy

Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model

Promiscuous expression of Epstein‐Barr virus genes in Burkitt's lymphoma from the central African country Malawi

Octa-guanidine Morpholino Restores Dystrophin Expression in Cardiac and Skeletal Muscles and Ameliorates Pathology in Dystrophic mdx Mice

In Vivo Evaluation of Dystrophin Exon Skipping in mdx Mice

BCL-2 expression in adult and embryonic non-haematopoietic tissues

Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic Mice

A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy

ISPD Overexpression Enhances Ribitol-Induced Glycosylation of α-Dystroglycan in Dystrophic FKRP Mutant Mice

Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic mdx Mice by Peptide-Conjugated Morpholino

bcl-2: Role in epithelial differentiation and oncogenesis

Systemic Delivery of Antisense Oligoribonucleotide Restores Dystrophin Expression in Body-Wide Skeletal Muscles

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