Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy

Protein Biomarker Quantification by Immunoaffinity Liquid Chromatography–Tandem Mass Spectrometry: Current State and Future Vision

(2020) Hendrik Neubert et al.   CLINICAL CHEMISTRY

Absolute Quantification of Dystrophin Protein in Human Muscle Biopsies using Parallel Reaction Monitoring (PRM)

(2019) Emily H. Canessa et al.   JOURNAL OF MASS SPECTROMETRY

Human FcRn Tissue Expression Profile and Half-Life in PBMCs

(2019) Yao-Yun Fan et al.   Biomolecules

Orthogonal analysis of dystrophin protein and mRNA as a surrogate outcome for drug development

(2019) Kitipong Uaesoontrachoon et al.   Biomarkers in Medicine

2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 – Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)

(2019) Steven Piccoli et al.   Bioanalysis

Targeted mass spectrometry enables robust quantification of FANCD2 mono-ubiquitination in response to DNA damage

(2018) Jeffrey R. Whiteaker et al.   DNA REPAIR

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

A novel high-throughput immunofluorescence analysis method for quantifying dystrophin intensity in entire transverse sections of Duchenne muscular dystrophy muscle biopsy samples

(2018) Valentina Sardone et al.   PLoS One

Use of capillary Western immunoassay (Wes) for quantification of dystrophin levels in skeletal muscle of healthy controls and individuals with Becker and Duchenne muscular dystrophy

(2018) Chantal Beekman et al.   PLoS One

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

(2017) Caroline Le Guiner et al.   Nature Communications

Quantitative Analysis of Human Neonatal Fc Receptor (FcRn) Tissue Expression in Transgenic Mice by Online Peptide Immuno-Affinity LC-HRMS

(2016) Yao-Yun Fan et al.   ANALYTICAL CHEMISTRY

Quantification of protein biomarkers in tissues: new capabilities with pellet digestion peptide immunoaffinity LC–MS/MS

(2016) Hendrik Neubert et al.   Bioanalysis

The Pathogenesis and Therapy of Muscular Dystrophies

(2015) Simon Guiraud et al.   Annual Review of Genomics and Human Genetics

The necessity of and strategies for improving confidence in the accuracy of western blots

(2014) Rajeshwary Ghosh et al.   Expert Review of Proteomics

Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients

(2014) Caroline Le Guiner et al.   MOLECULAR THERAPY

Dystrophin quantification: Biological and translational research implications

(2014) K. Anthony et al.   NEUROLOGY

Characterization of Dystrophin Deficient Rats: A New Model for Duchenne Muscular Dystrophy

(2014) Thibaut Larcher et al.   PLoS One

Highly Specific and Sensitive Measurements of Human and Monkey Interleukin 21 Using Sequential Protein and Tryptic Peptide Immunoaffinity LC-MS/MS

(2013) Joe Palandra et al.   ANALYTICAL CHEMISTRY

Dystrophin levels and clinical severity in Becker muscular dystrophy patients

(2013) J. C. van den Bergen et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy

(2013) Jin-Hong Shin et al.   MOLECULAR THERAPY

Sequential Protein and Peptide Immunoaffinity Capture for Mass Spectrometry-Based Quantification of Total Human β-Nerve Growth Factor

(2012) Hendrik Neubert et al.   ANALYTICAL CHEMISTRY

Evidence-based path to newborn screening for duchenne muscular dystrophy

(2012) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Multiplexed quantification of estrogen receptor and HER2/Neu in tissue and cell lysates by peptide immunoaffinity enrichment mass spectrometry

(2012) Regine M. Schoenherr et al.   PROTEOMICS

Online High-Flow Peptide Immunoaffinity Enrichment and Nanoflow LC-MS/MS: Assay Development for Total Salivary Pepsin/Pepsinogen

(2010) H. Neubert et al.   CLINICAL CHEMISTRY

A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in Dogs

(2008) Yongping Yue et al.   MOLECULAR THERAPY