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Engineered transfer RNAs for suppression of premature termination codons

Nature communications, 2019-02, Vol.10 (1), p.822-822, Article 822

2019

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Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAs

Molecular therapy. Nucleic acids, 2022-06, Vol.28, p.685-701

2022

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A cystic fibrosis gene editing approach that is on target

Molecular therapy. Nucleic acids, 2024-06, Vol.35 (2), p.102197-102197, Article 102197

2024

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Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

Science (American Association for the Advancement of Science), 2009-07, Vol.325 (5938), p.336-339

2009

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β11-12 linker isomerization governs acid-sensing ion channel desensitization and recovery

eLife, 2020-02, Vol.9

2020

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Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

The Journal of clinical investigation, 2007-12, Vol.117 (12), p.3952-3957

2007

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Cross-kingdom auxiliary subunit modulation of a voltage-gated sodium channel

The Journal of biological chemistry, 2018-04, Vol.293 (14), p.4981-4992

2018

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Altered mRNA splicing of the skeletal muscle ryanodine receptor and sarcoplasmic/endoplasmic reticulum Ca2+-ATPase in myotonic dystrophy type 1

Human molecular genetics, 2005-08, Vol.14 (15), p.2189-2200

2005

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Genetic ablation of complement C3 attenuates muscle pathology in dysferlin-deficient mice

The Journal of clinical investigation, 2010-12, Vol.120 (12), p.4366-4374

2010

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Allele-specific gene silencing in two mouse models of autosomal dominant skeletal myopathy

PloS one, 2012-11, Vol.7 (11), p.e49757-e49757

2012

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Cellular encoding of Cy dyes for single-molecule imaging

eLife, 2016-12, Vol.5

2016

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Heat- and anesthesia-induced malignant hyperthermia in an RyR1 knock-in mouse

The FASEB journal, 2006-02, Vol.20 (2), p.329-330

2006

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Atomic mutagenesis in ion channels with engineered stoichiometry

eLife, 2016-10, Vol.5

2016

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Therapeutic promise of engineered nonsense suppressor tRNAs

Wiley interdisciplinary reviews. RNA, 2021-07, Vol.12 (4), p.e1641-n/a

2021

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Sarcolemmal-restricted localization of functional ClC-1 channels in mouse skeletal muscle

The Journal of general physiology, 2010-12, Vol.136 (6), p.597-613

2010

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Photomodulation of the ASIC1a acidic pocket destabilizes the open state

Protein science, 2023-11, Vol.32 (11), p.e4800-n/a

2023

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Verapamil mitigates chloride and calcium bi-channelopathy in a myotonic dystrophy mouse model

The Journal of clinical investigation, 2024-01, Vol.134 (1), p.1-14

2024

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Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

Journal of cystic fibrosis, 2023-03, Vol.22 (Suppl 1), p.S39-S44

2023

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Chloride channelopathy in myotonic dystrophy resulting from loss of posttranscriptional regulation for CLCN1

American Journal of Physiology: Cell Physiology, 2007-04, Vol.292 (4), p.C1291-C1297

2007

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Correction of CIC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

The Journal of clinical investigation, 2007-12, Vol.117 (12), p.3952

2007

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Orthogonality of Pyrrolysine tRNA in the Xenopus oocyte

Scientific reports, 2018-03, Vol.8 (1), p.5166-6, Article 5166

2018

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Mouse fukutin deletion impairs dystroglycan processing and recapitulates muscular dystrophy

The Journal of clinical investigation, 2012-09, Vol.122 (9), p.3330-3342

2012

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Muscle chloride channel dysfunction in two mouse models of myotonic dystrophy

The Journal of general physiology, 2007-01, Vol.129 (1), p.79-94

2007

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Alternative splicing of RyR1 alters the efficacy of skeletal EC coupling

Cell calcium (Edinburgh), 2009-03, Vol.45 (3), p.264-274

2009

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High termination activity negatively influences nonsense suppression

Biophysical journal, 2024-02, Vol.123 (3), p.320a-320a

2024

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Engineered transfer RNAs for suppression of premature termination codons

Efficient suppression of endogenous CFTR nonsense mutations using anticodon-engineered transfer RNAs

A cystic fibrosis gene editing approach that is on target

Reversal of RNA Dominance by Displacement of Protein Sequestered on Triplet Repeat RNA

β11-12 linker isomerization governs acid-sensing ion channel desensitization and recovery

Correction of ClC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

Cross-kingdom auxiliary subunit modulation of a voltage-gated sodium channel

Altered mRNA splicing of the skeletal muscle ryanodine receptor and sarcoplasmic/endoplasmic reticulum Ca2+-ATPase in myotonic dystrophy type 1

Genetic ablation of complement C3 attenuates muscle pathology in dysferlin-deficient mice

Allele-specific gene silencing in two mouse models of autosomal dominant skeletal myopathy

Cellular encoding of Cy dyes for single-molecule imaging

Heat- and anesthesia-induced malignant hyperthermia in an RyR1 knock-in mouse

Atomic mutagenesis in ion channels with engineered stoichiometry

Therapeutic promise of engineered nonsense suppressor tRNAs

Sarcolemmal-restricted localization of functional ClC-1 channels in mouse skeletal muscle

Photomodulation of the ASIC1a acidic pocket destabilizes the open state

Verapamil mitigates chloride and calcium bi-channelopathy in a myotonic dystrophy mouse model

Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis

Chloride channelopathy in myotonic dystrophy resulting from loss of posttranscriptional regulation for CLCN1

Correction of CIC-1 splicing eliminates chloride channelopathy and myotonia in mouse models of myotonic dystrophy

Orthogonality of Pyrrolysine tRNA in the Xenopus oocyte

Mouse fukutin deletion impairs dystroglycan processing and recapitulates muscular dystrophy

Muscle chloride channel dysfunction in two mouse models of myotonic dystrophy

Alternative splicing of RyR1 alters the efficacy of skeletal EC coupling

High termination activity negatively influences nonsense suppression

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