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Open access

Research article

First published 2022-01-04

Pre Clinical Assessment of AAVrh74.MCK.GNE Viral Vector Therapeutic Potential: Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy

Stella Mitrani-Rosenbaum, Lena Yakovlev, Michal Becker Cohen [...]

Journal of Neuromuscular Diseases

Free access

Research article

First published 2022-01-04

Visualizing Muscle Sialic Acid Expression in the GNE D207V Tg Gne -/- Cmah -/- Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches

Kelly E. Crowe, Deborah A. Zygmunt, Kristin Heller [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2024

Induced Muscle and Liver Absence of Gne in Postnatal Mice Does Not Result in Structural or Functional Muscle Impairment

Avi Harazi, Lena Yakovlev, Nili Ilouz [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2020

Upregulation of Hallmark Muscle Genes Protects Gne M743T/M743T Mutated Knock-In Mice From Kidney and Muscle Phenotype

Hadar Benyamini, Yehuda Kling, Lena Yakovlev [...]

Journal of Neuromuscular Diseases

Open access

Research article

First published 2025-3

Ultra-Orphan drug development for GNE Myopathy: A synthetic literature review and meta-analysis

Naoki Suzuki, Madoka Mori-Yoshimura, Ichizo Nishino [...]

Journal of Neuromuscular Diseases

Open access

Review article

First published 2009

Hereditary Inclusion Body Myopathy (HIBM2)

Chris M. Jay, Nick Levonyak, Gregory Nemunaitis [...]

Gene Regulation and Systems Biology

Retricted access

Research article

First published 2012-05

Serum Neural Cell Adhesion Molecule Is Hyposialylated in Hereditary Inclusion Body Myopathy

Yadira Valles-Ayoub, Saghi Esfandiarifard, Pedram Sinai [...]

Genetic Testing and Molecular Biomarkers

Search results

Pre Clinical Assessment of AAVrh74.MCK.GNE Viral Vector Therapeutic Potential: Robust Activity Despite Lack of Consistent Animal Model for GNE Myopathy

Visualizing Muscle Sialic Acid Expression in the GNE D207V Tg Gne -/- Cmah -/- Model of GNE Myopathy: A Comparison of Dietary and Gene Therapy Approaches

Induced Muscle and Liver Absence of Gne in Postnatal Mice Does Not Result in Structural or Functional Muscle Impairment

Upregulation of Hallmark Muscle Genes Protects Gne M743T/M743T Mutated Knock-In Mice From Kidney and Muscle Phenotype

Ultra-Orphan drug development for GNE Myopathy: A synthetic literature review and meta-analysis

Hereditary Inclusion Body Myopathy (HIBM2)

Serum Neural Cell Adhesion Molecule Is Hyposialylated in Hereditary Inclusion Body Myopathy