Duchenne muscular dystrophy drug discovery – the application of utrophin promoter activation screening

A Method of Utrophin Up-Regulation through RNAi-Mediated Knockdown of the Transcription Factor EN1

(2013) Q Wang et al.   JOURNAL OF INTERNATIONAL MEDICAL RESEARCH

AMPK Activation Stimulates Autophagy and Ameliorates Muscular Dystrophy in the mdx Mouse Diaphragm

(2012) Marion Pauly et al.   AMERICAN JOURNAL OF PATHOLOGY

Evidence-based path to newborn screening for duchenne muscular dystrophy

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

Impacts of Dystrophin and Utrophin Domains on Actin Structural Dynamics: Implications for Therapeutic Design

(2012) Ava Yun Lin et al.   JOURNAL OF MOLECULAR BIOLOGY

Canine models of Duchenne muscular dystrophy and their use in therapeutic strategies

(2012) Joe N. Kornegay et al.   MAMMALIAN GENOME

Rescue of Dystrophic Skeletal Muscle by PGC-1α Involves a Fast to Slow Fiber Type Shift in the mdx Mouse

(2012) Joshua T. Selsby et al.   PLoS One

Restoring Dystrophin Expression in Duchenne Muscular Dystrophy Muscle

(2011) Eric P. Hoffman et al.   AMERICAN JOURNAL OF PATHOLOGY

Discovery and SAR of 2-arylbenzotriazoles and 2-arylindazoles as potential treatments for Duchenne muscular dystrophy

(2011) Olivier De Moor et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Impending therapies for Duchenne muscular dystrophy

(2011) Terence A. Partridge   CURRENT OPINION IN NEUROLOGY

Chronic AMPK activation evokes the slow, oxidative myogenic program and triggers beneficial adaptations in mdx mouse skeletal muscle

(2011) V. Ljubicic et al.   HUMAN MOLECULAR GENETICS

TAT-μUtrophin mitigates the pathophysiology of dystrophin and utrophin double-knockout mice

(2011) Jarrod A. Call et al.   JOURNAL OF APPLIED PHYSIOLOGY

Mammalian Models of Duchenne Muscular Dystrophy: Pathological Characteristics and Therapeutic Applications

(2011) Akinori Nakamura et al.   JOURNAL OF BIOMEDICINE AND BIOTECHNOLOGY

Discovery of 2-Arylbenzoxazoles as Upregulators of Utrophin Production for the Treatment of Duchenne Muscular Dystrophy

(2011) Daniel R. Chancellor et al.   JOURNAL OF MEDICINAL CHEMISTRY

Current Status of Pharmaceutical and Genetic Therapeutic Approaches to Treat DMD

(2011) Christophe Pichavant et al.   MOLECULAR THERAPY

Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

(2011) Sebahattin Cirak et al.   MOLECULAR THERAPY

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

(2011) Dawn E Bowles et al.   MOLECULAR THERAPY

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

(2011) Nathalie M. Goemans et al.   NEW ENGLAND JOURNAL OF MEDICINE

Daily Treatment with SMTC1100, a Novel Small Molecule Utrophin Upregulator, Dramatically Reduces the Dystrophic Symptoms in the mdx Mouse

(2011) Jonathon M. Tinsley et al.   PLoS One

Translational Regulation of Utrophin by miRNAs

(2011) Utpal Basu et al.   PLoS One

Drug Discovery for Duchenne Muscular Dystrophy via Utrophin Promoter Activation Screening

(2011) Catherine Moorwood et al.   PLoS One

Drug screening in a zebrafish model of Duchenne muscular dystrophy

(2011) G. Kawahara et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Stem Cell Therapies to Treat Muscular Dystrophy

(2010) Mirella Meregalli et al.   BIODRUGS

Flt-1 haploinsufficiency ameliorates muscular dystrophy phenotype by developmentally increased vasculature in mdx mice

(2010) Mayank Verma et al.   HUMAN MOLECULAR GENETICS

The utrophin A 5'-UTR drives cap-independent translation exclusively in skeletal muscles of transgenic mice and interacts with eEF1A2

(2010) P. Miura et al.   HUMAN MOLECULAR GENETICS

Change in Natural History of Duchenne Muscular Dystrophy With Long-term Corticosteroid Treatment: Implications for Management

(2010) Richard T. Moxley et al.   JOURNAL OF CHILD NEUROLOGY

RhoA leads to up-regulation and relocalization of utrophin in muscle fibers

(2009) Cécile Gauthier-Rouvière et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Differential Expression of Utrophin-A and -B Promoters in the Central Nervous System (CNS) of Normal and DystrophicmdxMice

(2009) Santhosh M. Baby et al.   BRAIN PATHOLOGY

Pharmacological activation of PPAR /  stimulates utrophin A expression in skeletal muscle fibers and restores sarcolemmal integrity in mature mdx mice

(2009) P. Miura et al.   HUMAN MOLECULAR GENETICS

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Novel activation domain derived from Che-1 cofactor coupled with the artificial protein Jazz drives utrophin upregulation

(2009) Agata Desantis et al.   NEUROMUSCULAR DISORDERS

Mammalian animal models for Duchenne muscular dystrophy

(2009) Raffaella Willmann et al.   NEUROMUSCULAR DISORDERS

Dystrophin and utrophin have distinct effects on the structural dynamics of actin

(2009) E. Prochniewicz et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Functional Substitution by TAT-Utrophin in Dystrophin-Deficient Mice

(2009) Kevin J. Sonnemann et al.   PLOS MEDICINE

Targeting Artificial Transcription Factors to the Utrophin A Promoter

(2008) Yifan Lu et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Downstream utrophin enhancer is required for expression of utrophin in skeletal muscle

(2008) Jun Tanihata et al.   JOURNAL OF GENE MEDICINE

Disability and survival in Duchenne muscular dystrophy

(2008) M Kohler et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Microutrophin Delivery Through rAAV6 Increases Lifespan and Improves Muscle Function in Dystrophic Dystrophin/Utrophin-deficient Mice

(2008) Guy L Odom et al.   MOLECULAR THERAPY

Gene-mediated Restoration of Normal Myofiber Elasticity in Dystrophic Muscles

(2008) Stefania Puttini et al.   MOLECULAR THERAPY

IRES-Mediated Translation of Utrophin A Is Enhanced by Glucocorticoid Treatment in Skeletal Muscle Cells

(2008) Pedro Miura et al.   PLoS One