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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Journal of cachexia, sarcopenia and muscle, 2020-06, Vol.11 (3), p.768-782

2020

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Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

Molecular therapy, 2018-06, Vol.26 (6), p.1529-1538

2018

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Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)

Journal of cachexia, sarcopenia and muscle, 2019-10, Vol.10 (5), p.1016-1026

2019

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Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model

Human molecular genetics, 2021-07, Vol.30 (15), p.1398-1412

2021

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Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy

Human gene therapy, 2022-09, Vol.33 (17-18), p.923

2022

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Systemic Pharmacotherapeutic Treatment of the ACTA1-MCM/FLExDUX4 Preclinical Mouse Model of FSHD

International journal of molecular sciences, 2024-07, Vol.25 (13), p.6994

2024

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Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon 17 Partially Addresses Fibrosis in D2.mdx Mice

International journal of molecular sciences, 2024-06, Vol.25 (11), p.6113

2024

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Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

International journal of molecular sciences, 2023-05, Vol.24 (9), p.8174

2023

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Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation

Methods in molecular biology (Clifton, N.J.), 2018, Vol.1828, p.343

2018

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Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1

Human molecular genetics, 2019-10, Vol.28 (19), p.3301-3308

2019

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Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression

Biomedicines, 2022-07, Vol.10 (7), p.1623

2022

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Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy

Human molecular genetics, 2019-05, Vol.28 (10), p.1694-1708

2019

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Expression of the Pro-Fibrotic Marker Periostin in a Mouse Model of Duchenne Muscular Dystrophy

Biomedicines, 2024-01, Vol.12 (1), p.216

2024

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Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Molecular therapy, 2015-08, Vol.23 (8), p.1341-1348

2015

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Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Molecular therapy. Nucleic acids, 2017-03, Vol.6, p.15-28

2017

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Efficient Expression of Igf-1 from Lentiviral Vectors Protects In Vitro but Does Not Mediate Behavioral Recovery of a Parkinsonian Lesion in Rats

Human gene therapy, 2015-11, Vol.26 (11), p.719

2015

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Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease

Human gene therapy, 2014-07, Vol.25 (7), p.631

2014

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BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

Molecular therapy. Nucleic acids, 2021-06, Vol.24, p.67-78

2021

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An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

Biomedicines, 2023-10, Vol.11 (10), p.2700

2023

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Intrastriatal Delivery of Integration-Deficient Lentiviral Vectors in a Rat Model of Parkinson's Disease

Methods in molecular biology (Clifton, N.J.), 2016, Vol.1448, p.175

2016

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Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides

Biomedicines, 2018-06, Vol.6 (3), p.74

2018

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Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment

Methods in molecular biology (Clifton, N.J.), 2022, Vol.2434, p.301

2022

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Antisense Oligonucleotide Targeting of 3'-UTR of mRNA for Expression Knockdown

Methods in molecular biology (Clifton, N.J.), 2018, Vol.1828, p.91

2018

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Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element

Human gene therapy, 2021-10, Vol.32 (19-20), p.1138

2021

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An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier Vectors and Antisense Oligonucleotides

Biomedicines, 2023-10, Vol.11 (10)

2023

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Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy

Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD)

Systemic antisense therapeutics inhibiting DUX4 expression ameliorates FSHD-like pathology in an FSHD mouse model

Improving Molecular and Histopathology in Diaphragm Muscle of the Double Transgenic ACTA1-MCM/FLExDUX4 Mouse Model of FSHD with Systemic Antisense Therapy

Systemic Pharmacotherapeutic Treatment of the ACTA1-MCM/FLExDUX4 Preclinical Mouse Model of FSHD

Targeted Antisense Oligonucleotide-Mediated Skipping of Murine Postn Exon 17 Partially Addresses Fibrosis in D2.mdx Mice

Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

Systemic Intravenous Administration of Antisense Therapeutics for Combinatorial Dystrophin and Myostatin Exon Splice Modulation

Established PABPN1 intranuclear inclusions in OPMD muscle can be efficiently reversed by AAV-mediated knockdown and replacement of mutant expanded PABPN1

Long-Term Systemic Treatment of a Mouse Model Displaying Chronic FSHD-like Pathology with Antisense Therapeutics That Inhibit DUX4 Expression

Pharmacological modulation of the ER stress response ameliorates oculopharyngeal muscular dystrophy

Expression of the Pro-Fibrotic Marker Periostin in a Mouse Model of Duchenne Muscular Dystrophy

Combination Antisense Treatment for Destructive Exon Skipping of Myostatin and Open Reading Frame Rescue of Dystrophin in Neonatal mdx Mice

Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

Efficient Expression of Igf-1 from Lentiviral Vectors Protects In Vitro but Does Not Mediate Behavioral Recovery of a Parkinsonian Lesion in Rats

Transgenic expression of human glial cell line-derived neurotrophic factor from integration-deficient lentiviral vectors is neuroprotective in a rodent model of Parkinson's disease

BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides

Intrastriatal Delivery of Integration-Deficient Lentiviral Vectors in a Rat Model of Parkinson's Disease

Targeting TGFβ Signaling to Address Fibrosis Using Antisense Oligonucleotides

Use of Small Animal Models for Duchenne and Parameters to Assess Efficiency upon Antisense Treatment

Antisense Oligonucleotide Targeting of 3'-UTR of mRNA for Expression Knockdown

Dose-Dependent Microdystrophin Expression Enhancement in Cardiac Muscle by a Cardiac-Specific Regulatory Element

An Induced Pluripotent Stem Cell-Derived Human Blood–Brain Barrier Vectors and Antisense Oligonucleotides

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