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RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement

Molecular therapy. Nucleic acids, 2019-04, Vol.15, p.12-25

2019

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Sirtuin inhibition protects from the polyalanine muscular dystrophy protein PABPN1

Human molecular genetics, 2008-07, Vol.17 (14), p.2108-2117

2008

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Soluble expanded PABPN1 promotes cell death in oculopharyngeal muscular dystrophy

Neurobiology of disease, 2007-06, Vol.26 (3), p.546-557

2007

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CRISPR/CAS9 system corrects PABPN1 mutation in oculopharyngeal muscular dystrophy

Journal of the neurological sciences, 2023-12, Vol.455, p.121728, Article 121728

2023

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Cytoplasmic Targeting of Mutant Poly(A)‐Binding Protein Nuclear 1 Suppresses Protein Aggregation and Toxicity in Oculopharyngeal Muscular Dystrophy

Traffic (Copenhagen, Denmark), 2005-09, Vol.6 (9), p.766-779

2005

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Oculopharyngeal muscular dystrophy: Recent advances in the understanding of the molecular pathogenic mechanisms and treatment strategies

Biochimica et biophysica acta, 2007-02, Vol.1772 (2), p.173-185

2007

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Involvement of the ubiquitin-proteasome pathway and molecular chaperones in oculopharyngeal muscular dystrophy

Human molecular genetics, 2003-10, Vol.12 (20), p.2609-2623

2003

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Automated design of hammerhead ribozymes and validation by targeting the PABPN1 gene transcript

Nucleic acids research, 2016-02, Vol.44 (4), p.e39

2016

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Evolutionary Design and Experimental Evaluation of Selective Hammerhead Ribozymes

2020 IEEE 20th International Conference on Bioinformatics and Bioengineering (BIBE), 2020, p.12-16

2020

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Molecular mechanism of enhanced uv-mutagenesis in the tk-deficient mutant subclone of friend mouse erythroleukaemia cells

Abu-Baker, Aida

2000

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Chapter 33 - Polyalanine and Polyglutamine Diseases: Possible Common Mechanisms?

Genetic Instabilities and Neurological Diseases, Second Edition, 2006, p.487-513

Second Edition, 2006

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P V.20 Isolation and analysis of spontaneous and UV-induced HPRT mutants of wild-type and thymidine kinase-deficient friend mouse erythroleukaemia (FEL) cells

Mutation research, 1997-09, Vol.379 (1), p.S39-S39

1997

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RNA-Based Therapy Utilizing Oculopharyngeal Muscular Dystrophy Transcript Knockdown and Replacement

Sirtuin inhibition protects from the polyalanine muscular dystrophy protein PABPN1

Soluble expanded PABPN1 promotes cell death in oculopharyngeal muscular dystrophy

CRISPR/CAS9 system corrects PABPN1 mutation in oculopharyngeal muscular dystrophy

Cytoplasmic Targeting of Mutant Poly(A)‐Binding Protein Nuclear 1 Suppresses Protein Aggregation and Toxicity in Oculopharyngeal Muscular Dystrophy

Oculopharyngeal muscular dystrophy: Recent advances in the understanding of the molecular pathogenic mechanisms and treatment strategies

Involvement of the ubiquitin-proteasome pathway and molecular chaperones in oculopharyngeal muscular dystrophy

Automated design of hammerhead ribozymes and validation by targeting the PABPN1 gene transcript

Evolutionary Design and Experimental Evaluation of Selective Hammerhead Ribozymes

Molecular mechanism of enhanced uv-mutagenesis in the tk-deficient mutant subclone of friend mouse erythroleukaemia cells

Chapter 33 - Polyalanine and Polyglutamine Diseases: Possible Common Mechanisms?

P V.20 Isolation and analysis of spontaneous and UV-induced HPRT mutants of wild-type and thymidine kinase-deficient friend mouse erythroleukaemia (FEL) cells

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