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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

Molecular therapy, 2012-02, Vol.20 (2), p.443-455

2012

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BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Blood, 2021-02, Vol.137 (6), p.763-774

2021

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Long-term follow-up after gene therapy for canavan disease

Science translational medicine, 2012-12, Vol.4 (165), p.165ra163

2012

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Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Human gene therapy, 2015-02, Vol.26 (2), p.69

2015

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Mechanistic insights of evaporation-induced actuation in supramolecular crystals

Nature materials, 2021-03, Vol.20 (3), p.403-409

2021

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Crystal structure and mechanistic investigation of the twister ribozyme

Nature chemical biology, 2014-09, Vol.10 (9), p.739

2014

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Polymeric peptide pigments with sequence-encoded properties

Science (American Association for the Advancement of Science), 2017-06, Vol.356 (6342), p.1064-1068

2017

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Melanin‐Inspired Chromophoric Microparticles Composed of Polymeric Peptide Pigments

Angewandte Chemie (International ed.), 2021-03, Vol.60 (14), p.7564-7569

International ed. in English, 2021

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Emergence of Cooperative Glucose-Binding Networks in Adaptive Peptide Systems

Journal of the American Chemical Society, 2023-05, Vol.145 (17), p.9800-9807

2023

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Dystrophin Immunity in Duchenne's Muscular Dystrophy

The New England journal of medicine, 2010-10, Vol.363 (15), p.1429-1437

2010

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The Structure of Sulfoindocarbocyanine 3 Terminally Attached to dsDNA via a Long, Flexible Tether

Biophysical journal, 2012-02, Vol.102 (3), p.561-568

2012

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A critical base pair in k-turns that confers folding characteristics and correlates with biological function

Nature communications, 2014-10, Vol.5 (1), p.5127-5127, Article 5127

2014

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Fluorescent RNA cytosine analogue - an internal probe for detailed structure and dynamics investigations

Scientific reports, 2017-05, Vol.7 (1), p.2393-8, Article 2393

2017

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Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

Nature biotechnology, 2010-01, Vol.28 (1), p.79-82

2010

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Author Correction: Mechanistic insights of evaporation-induced actuation in supramolecular crystals

Nature materials, 2021-03, Vol.20 (3), p.434-434

2021

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The Impact of Tyrosine Iodination on the Aggregation and Cleavage Kinetics of MMP-9-Responsive Peptide Sequences

ACS biomaterials science & engineering, 2022-02, Vol.8 (2), p.579-587

2022

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Unbiased Discovery of Dynamic Peptide‐ATP Complexes

ChemSystemsChem, 2019-07, Vol.1 (1-2), p.7-11

2019

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Melanin‐Inspired Chromophoric Microparticles Composed of Polymeric Peptide Pigments

Angewandte Chemie, 2021-03, Vol.133 (14), p.7642-7647

2021

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Unbiased Discovery of Dynamic Peptide‐ATP Complexes

ChemSystemsChem, 2019-07, Vol.1 (1-2), p.2-2

2019

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Synthesis of novel tetrazole C5-linked C0- and C2-ribonucleoside phosphoramidites using MePOM and POM groups for probing RNA catalysis

Tetrahedron letters, 2012-10, Vol.53 (44), p.5891-5894

2012

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Novel role for aspartoacylase in regulation of BDNF and timing of postnatal oligodendrogenesis

Journal of neuroscience research, 2006-07, Vol.84 (1), p.151-169

2006

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Assessment of hippocampal adeno-associated viral vector gene delivery via frameless stereotaxis in a nonhuman primate

Stereotactic and functional neurosurgery, 2011-01, Vol.89 (5), p.275

2011

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Viral-based gene transfer to the mammalian CNS for functional genomic studies

Trends in Neurosciences, 2001-12, Vol.24 (12), p.706-712

2001

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Physical agent modalities: a position paper

The American journal of occupational therapy, 2008-11, Vol.62 (6), p.691

2008

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Prediction of adeno-associated virus neutralizing antibody activity for clinical application

Gene therapy, 2015-12, Vol.22 (12), p.984-992

2015

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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression

Long-term follow-up after gene therapy for canavan disease

Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Mechanistic insights of evaporation-induced actuation in supramolecular crystals

Crystal structure and mechanistic investigation of the twister ribozyme

Polymeric peptide pigments with sequence-encoded properties

Melanin‐Inspired Chromophoric Microparticles Composed of Polymeric Peptide Pigments

Emergence of Cooperative Glucose-Binding Networks in Adaptive Peptide Systems

Dystrophin Immunity in Duchenne's Muscular Dystrophy

The Structure of Sulfoindocarbocyanine 3 Terminally Attached to dsDNA via a Long, Flexible Tether

A critical base pair in k-turns that confers folding characteristics and correlates with biological function

Fluorescent RNA cytosine analogue - an internal probe for detailed structure and dynamics investigations

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

Author Correction: Mechanistic insights of evaporation-induced actuation in supramolecular crystals

The Impact of Tyrosine Iodination on the Aggregation and Cleavage Kinetics of MMP-9-Responsive Peptide Sequences

Unbiased Discovery of Dynamic Peptide‐ATP Complexes

Melanin‐Inspired Chromophoric Microparticles Composed of Polymeric Peptide Pigments

Unbiased Discovery of Dynamic Peptide‐ATP Complexes

Synthesis of novel tetrazole C5-linked C0- and C2-ribonucleoside phosphoramidites using MePOM and POM groups for probing RNA catalysis

Novel role for aspartoacylase in regulation of BDNF and timing of postnatal oligodendrogenesis

Assessment of hippocampal adeno-associated viral vector gene delivery via frameless stereotaxis in a nonhuman primate

Viral-based gene transfer to the mammalian CNS for functional genomic studies

Physical agent modalities: a position paper

Prediction of adeno-associated virus neutralizing antibody activity for clinical application

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