Related references
Note: Only part of the references are listed.Rebalancing expression of HMGB1 redox isoforms to counteract muscular dystrophy
Giorgia Careccia et al.
SCIENCE TRANSLATIONAL MEDICINE (2021)
Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery
Marcello Carotti et al.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)
Intraperitoneal Route of Drug Administration: Should it Be Used in Experimental Animal Studies?
Abdullah Al Shoyaib et al.
PHARMACEUTICAL RESEARCH (2020)
CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine
Miqueias Lopes-Pacheco
FRONTIERS IN PHARMACOLOGY (2020)
New genotype-phenotype correlations in a large European cohort of patients with sarcoglycanopathy
Jorge Alonso-Perez et al.
BRAIN (2020)
Repairing folding-defective α-sarcoglycan mutants by CFTR correctors, a potential therapy for limb-girdle muscular dystrophy 2D
Marcello Carotti et al.
HUMAN MOLECULAR GENETICS (2018)
229th ENMC international workshop: Limb girdle muscular dystrophies - Nomenclature and reformed classification Naarden, the Netherlands, 17-19 March 2017
Volker Straub et al.
NEUROMUSCULAR DISORDERS (2018)
Different outcome of sarcoglycan missense mutation between human and mouse
Sara F. Henriques et al.
PLOS ONE (2018)
Application of Real-Time Quantitative PCR to Detect Mink Circovirus in Naturally and Experimentally Infected Minks
Xingyang Cui et al.
FRONTIERS IN MICROBIOLOGY (2018)
Emerging therapeutic strategies for sarcoglycanopathy
Marcello Carotti et al.
EXPERT OPINION ON ORPHAN DRUGS (2017)
Correcting CFTR folding defects by small-molecule correctors to cure cystic fibrosis
Marjolein Mijnders et al.
CURRENT OPINION IN PHARMACOLOGY (2017)
The sarcoglycan complex in skeletal muscle
Hakan Tarakci et al.
FRONTIERS IN BIOSCIENCE-LANDMARK (2016)
Rescue of defective ATP8B1 trafficking by CFTR correctors as a therapeutic strategy for familial intrahepatic cholestasis
Wendy L. van der Woerd et al.
JOURNAL OF HEPATOLOGY (2016)
Pathogenesis, clinical features and diagnosis of sarcoglycanopathies
Corrado Angelini et al.
EXPERT OPINION ON ORPHAN DRUGS (2016)
Rescuing Trafficking Mutants of the ATP-binding Cassette Protein, ABCA4, with Small Molecule Correctors as a Treatment for Stargardt Eye Disease
Inna Sabirzhanova et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2015)
Unveiling the degradative route of the V247M α-sarcoglycan mutant responsible for LGMD-2D
Elisa Bianchini et al.
HUMAN MOLECULAR GENETICS (2014)
Correctors of the basic trafficking defect of the mutant F508del-CFTR that causes cystic fibrosis
Veronique Birault et al.
CURRENT OPINION IN CHEMICAL BIOLOGY (2013)
Compounds that correct F508del-CFTR trafficking can also correct other protein trafficking diseases: an in vitro study using cell lines
Heidi M. Sampson et al.
ORPHANET JOURNAL OF RARE DISEASES (2013)
Rescue of Sarcoglycan Mutations by Inhibition of Endoplasmic Reticulum Quality Control is Associated with Minimal Structural Modifications
Tayebeh Soheili et al.
HUMAN MUTATION (2012)
Functional Fluorescently Labeled Bithiazole ΔF508-CFTR Corrector Imaged in Whole Body Slices in Mice
Holly R. Davison et al.
BIOCONJUGATE CHEMISTRY (2011)
Cystic Fibrosis Transmembrane Conductance Regulator Intracellular Processing, Trafficking, and Opportunities for Mutation-Specific Treatment
Mark P. Rogan et al.
CHEST (2011)
In vivo tissue engineering of functional skeletal muscle by freshly isolated satellite cells embedded in a photopolymerizable hydrogel
Carlo Alberto Rossi et al.
FASEB JOURNAL (2011)
Eccentric contractions lead to myofibrillar dysfunction in muscular dystrophy
Bert Blaauw et al.
JOURNAL OF APPLIED PHYSIOLOGY (2010)
MUSCLE WEAKNESS AND ATROPHY ARE ASSOCIATED WITH DECREASED REGENERATIVE CAPACITY AND CHANGES IN MTOR SIGNALING IN SKELETAL MUSCLES OF VENERABLE (18-24-MONTH-OLD) DYSTROPHIC MDX MICE
E. Mouisel et al.
MUSCLE & NERVE (2010)
Sarcoglycanopathies: molecular pathogenesis and therapeutic prospects
Dorianna Sandona et al.
EXPERT REVIEWS IN MOLECULAR MEDICINE (2009)
Renal toxicity of therapeutic drugs
R. John et al.
JOURNAL OF CLINICAL PATHOLOGY (2009)
Histological patterns in drug-induced liver disease
R. Ramachandran et al.
JOURNAL OF CLINICAL PATHOLOGY (2009)
Frequency of LGMD gene mutations in Italian patients with distinct clinical phenotypes
M. Fanin et al.
NEUROLOGY (2009)
Inhibition of proteasome activity promotes the correct localization of disease-causing α-sarcoglycan mutants in HEK-293 cells constitutively expressing β-, γ-, and δ-sarcoglycan
Stefano Gastaldello et al.
AMERICAN JOURNAL OF PATHOLOGY (2008)
Construction and analysis of compact muscle-specific promoters for AAV vectors
B. Wang et al.
GENE THERAPY (2008)
A common disease-associated missense mutation in alpha-sarcoglycan fails to cause muscular dystrophy in mice
Kazuhiro Kobuke et al.
HUMAN MOLECULAR GENETICS (2008)
Mannosidase I inhibition rescues the human α-sarcoglycan R77C recurrent mutation
Marc Bartoli et al.
HUMAN MOLECULAR GENETICS (2008)
Clinical, molecular, and protein correlations in a large sample of genetically diagnosed Italian limb girdle muscular dystrophy patients
Michela Guglieri et al.
HUMAN MUTATION (2008)
Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D
Christina A. Pacak et al.
MOLECULAR THERAPY (2007)
Processing and assembly of the dystrophin glycoprotein complex
Michael J. Allikian et al.
TRAFFIC (2007)
The β-δ-core of sarcoglycan is essential for deposition at the plasma membrane
Romesh A. Draviam et al.
MUSCLE & NERVE (2006)
Specific assembly pathway of sarcoglycans is dependent on beta- and delta-sarcoglycan
WX Shi et al.
MUSCLE & NERVE (2004)
Characterization of the ATP-hydrolysing activity of α-sarcoglycan
D Sandonà et al.
BIOCHEMICAL JOURNAL (2004)
A principal role for the proteasome in endoplasmic reticulum-associated degradation of misfolded intracellular cystic fibrosis transmembrane conductance regulator
MS Gelman et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2002)
Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice
V Allamand et al.
GENE THERAPY (2000)
Sarcoglycanopathies in Dutch patients with autosomal recessive limb girdle muscular dystrophy
HB Ginjaar et al.
JOURNAL OF NEUROLOGY (2000)
Share Paper
