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Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy

Pharmaceutics, 2022-10, Vol.14 (10), p.2129

2022

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The protective mutation A673T in amyloid precursor protein gene decreases Aβ peptides production for 14 forms of Familial Alzheimer's Disease in SH-SY5Y cells

PloS one, 2020-12, Vol.15 (12), p.e0237122-e0237122

2020

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Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy

International journal of molecular sciences, 2022-05, Vol.23 (11), p.6160

2022

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Prime Editing for Human Gene Therapy: Where Are We Now?

Cells (Basel, Switzerland), 2023-02, Vol.12 (4), p.536

2023

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Small-molecule inhibitors of proteasome increase CjCas9 protein stability

PloS one, 2023-01, Vol.18 (1), p.e0280353-e0280353

2023

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CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing

Gene therapy, 2022-04, Vol.29 (3-4), p.171-177

2022

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Limb-Girdle Muscular Dystrophies Classification and Therapies

Journal of clinical medicine, 2023-07, Vol.12 (14), p.4769

2023

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A Comment on “Muscle Xenografts Reproduce Key Molecular Features of Facioscapulohumeral Muscular Dystrophy”: What Is New and What Has Already been Done and Reported but Was Not Quoted?

Cell transplantation, 2020-01, Vol.29, p.96368972093912-963689720939120

2020

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Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

Molecular therapy, 2013-09, Vol.21 (9), p.1718-1726

2013

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Portrait of Dysferlinopathy: Diagnosis and Development of Therapy

Journal of clinical medicine, 2023-09, Vol.12 (18), p.6011

2023

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CRISPR-Cas9 delivery strategies with engineered extracellular vesicles

Molecular therapy. Nucleic acids, 2023-12, Vol.34, p.102040-102040, Article 102040

2023

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Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy

Frontiers in medicine, 2022-03, Vol.9, p.859930-859930

2022

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Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy

Cells (Basel, Switzerland), 2023-12, Vol.13 (1), p.31

2023

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Nonfunctional mutant Wrn protein leads to neurological deficits, neuronal stress, microglial alteration, and immune imbalance in a mouse model of Werner syndrome

Brain, behavior, and immunity, 2018-10, Vol.73, p.450-469

2018

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Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene

Molecular therapy. Nucleic acids, 2022-12, Vol.30, p.272-285

2022

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Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

Molecular therapy, 2011-05, Vol.19 (5), p.830-840

2011

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Clarifying Misconceptions About Myoblast Transplantation in Myology

Molecular therapy, 2014-05, Vol.22 (5), p.897-898

2014

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Emerging Perspectives on Prime Editor Delivery to the Brain

Pharmaceuticals (Basel, Switzerland), 2024-06, Vol.17 (6), p.763

2024

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Base editing strategy for insertion of the A673T mutation in the APP gene to prevent the development of AD in vitro

Molecular therapy. Nucleic acids, 2021-06, Vol.24, p.253-263

2021

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Improvements of nuclease and nickase gene modification techniques for the treatment of genetic diseases

Frontiers in genome editing, 2022-07, Vol.4, p.892769-892769

2022

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Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160

Molecular therapy. Nucleic acids, 2017-03, Vol.6, p.68-79

2017

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Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

Molecular therapy. Nucleic acids, 2016, Vol.5 (1), p.e283-e283, Article e283

2016

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Macrophages improve survival, proliferation and migration of engrafted myogenic precursor cells into MDX skeletal muscle

PloS one, 2012-10, Vol.7 (10), p.e46698-e46698

2012

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Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotype

Genes, 2023-08, Vol.14 (8), p.1654

2023

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Losartan Enhances the Success of Myoblast Transplantation

Cell transplantation, 2012-01, Vol.21 (1), p.139-152

2012

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Delivery of RNAs to Specific Organs by Lipid Nanoparticles for Gene Therapy

The protective mutation A673T in amyloid precursor protein gene decreases Aβ peptides production for 14 forms of Familial Alzheimer's Disease in SH-SY5Y cells

Prime Editing Permits the Introduction of Specific Mutations in the Gene Responsible for Duchenne Muscular Dystrophy

Prime Editing for Human Gene Therapy: Where Are We Now?

Small-molecule inhibitors of proteasome increase CjCas9 protein stability

CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing

Limb-Girdle Muscular Dystrophies Classification and Therapies

A Comment on “Muscle Xenografts Reproduce Key Molecular Features of Facioscapulohumeral Muscular Dystrophy”: What Is New and What Has Already been Done and Reported but Was Not Quoted?

Reading Frame Correction by Targeted Genome Editing Restores Dystrophin Expression in Cells From Duchenne Muscular Dystrophy Patients

Portrait of Dysferlinopathy: Diagnosis and Development of Therapy

CRISPR-Cas9 delivery strategies with engineered extracellular vesicles

Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy

Successful Correction by Prime Editing of a Mutation in the RYR1 Gene Responsible for a Myopathy

Nonfunctional mutant Wrn protein leads to neurological deficits, neuronal stress, microglial alteration, and immune imbalance in a mouse model of Werner syndrome

Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene

Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

Clarifying Misconceptions About Myoblast Transplantation in Myology

Emerging Perspectives on Prime Editor Delivery to the Brain

Base editing strategy for insertion of the A673T mutation in the APP gene to prevent the development of AD in vitro

Improvements of nuclease and nickase gene modification techniques for the treatment of genetic diseases

Increased Expression of Laminin Subunit Alpha 1 Chain by dCas9-VP160

Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

Macrophages improve survival, proliferation and migration of engrafted myogenic precursor cells into MDX skeletal muscle

Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotype

Losartan Enhances the Success of Myoblast Transplantation

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