4.6 Review

Myotonic dystrophy: Therapeutic strategies for the future

Related references

Note: Only part of the references are listed.
Article Clinical Neurology

A phase I/II trial of MYO-029 in adult subjects with muscular dystrophy

Kathryn R. Wagner et al.

ANNALS OF NEUROLOGY (2008)

Article Biotechnology & Applied Microbiology

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice

Natee Jearawiriyapaisarn et al.

MOLECULAR THERAPY (2008)

Article Multidisciplinary Sciences

Genetic and Chemical Modifiers of a CUG Toxicity Model in Drosophila

Amparo Garcia-Lopez et al.

PLOS ONE (2008)

Article Multidisciplinary Sciences

Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors

Amanda M. Haidet et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Multidisciplinary Sciences

Expanded CTG repeats within the DMPK 3′ UTR causes severe skeletal muscle wasting in an inducible mouse model for myotonic dystrophy

James P. Orengo et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Medicine, General & Internal

Local dystrophin restoration with antisense oligonucleotide PRO051

Judith C. van Deutekom et al.

NEW ENGLAND JOURNAL OF MEDICINE (2007)

Review Biochemistry & Molecular Biology

Design and implementation of cell-based assays to model human disease

Jeremy O. Jones et al.

ACS CHEMICAL BIOLOGY (2007)

Article Biochemistry & Molecular Biology

A small molecule microarray platform to select RNA internal loop-ligand interactions

Jessica L. Childs-Disney et al.

ACS CHEMICAL BIOLOGY (2007)

Article Biochemistry & Molecular Biology

Increased steady-state in levels of CUGBP1 in myotonic dystrophy 1 are due to PKC-mediated hyperphosphorylation

N. Muge Kuyumcu-Martinez et al.

MOLECULAR CELL (2007)

Article Medicine, Research & Experimental

Elevation of RNA-binding protein CUGBP1 is an early event in an inducible heart-specific mouse model of myotonic dystrophy

Guey-Shin Wang et al.

JOURNAL OF CLINICAL INVESTIGATION (2007)

Article Biochemistry & Molecular Biology

Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos

Paul A. Morcos

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2007)

Article Clinical Neurology

Ribonuclear foci at the neuromuscular junction in myotonic dystrophy type 1

T. M. Wheeler et al.

NEUROMUSCULAR DISORDERS (2007)

Article Biochemistry & Molecular Biology

Ribonuclease dicer cleaves triplet repeat hairpins into shorter repeats that silence specific targets

Jacelk Krol et al.

MOLECULAR CELL (2007)

Article Physiology

Muscle chloride channel dysfunction in two mouse models of myotonic dystrophy

John D. Lueck et al.

JOURNAL OF GENERAL PHYSIOLOGY (2007)

Article Biochemistry & Molecular Biology

A bichromatic fluorescent reporter for cell-based screens of alternative splicing

James P. Orengo et al.

NUCLEIC ACIDS RESEARCH (2006)

Review Biochemistry & Molecular Biology

RNA-dominant diseases

Robert J. Osborne et al.

HUMAN MOLECULAR GENETICS (2006)

Article Biochemistry & Molecular Biology

Functional and morphological recovery of dystrophic muscles in mice treated with deacetylase inhibitors

G. C. Minetti et al.

NATURE MEDICINE (2006)

Article Genetics & Heredity

Reversible model of RNA toxicity and cardiac conduction defects in myotonic dystrophy

Mani S. Mahadevan et al.

NATURE GENETICS (2006)

Article Multidisciplinary Sciences

Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy

Rahul N. Kanadia et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)

Article Biochemistry & Molecular Biology

Failure of MBNL1-dependent post-natal splicing transitions in myotonic dystrophy

Xiaoyan Lin et al.

HUMAN MOLECULAR GENETICS (2006)

Article Biochemistry & Molecular Biology

Biologically active molecules that reduce polyglutamine aggregation and toxicity

Urvee A. Desai et al.

HUMAN MOLECULAR GENETICS (2006)

Article Biochemistry & Molecular Biology

MBNL1 and CUGBP1 modify expanded CUG-induced toxicity in a Drosophila model of myotonic dystrophy type 1

Maria de Haro et al.

HUMAN MOLECULAR GENETICS (2006)

Article Endocrinology & Metabolism

Myofibrillar protein synthesis in myostatin-deficient mice

S Welle et al.

AMERICAN JOURNAL OF PHYSIOLOGY-ENDOCRINOLOGY AND METABOLISM (2006)

Article Biochemistry & Molecular Biology

MBNL1 is the primary determinant of focus formation and aberrant insulin receptor splicing in DM1

W Dansithong et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2005)

Article Multidisciplinary Sciences

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

QL Lu et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)

Article Biochemistry & Molecular Biology

Muscleblind proteins regulate alternative splicing

TH Ho et al.

EMBO JOURNAL (2004)

Article Biochemistry & Molecular Biology

Systemic delivery of genes to striated muscles using adeno-associated viral vectors

P Gregorevic et al.

NATURE MEDICINE (2004)

Article Medicine, General & Internal

Brief report -: Myostatin mutation associated with gross muscle hypertrophy in a child

M Schuelke et al.

NEW ENGLAND JOURNAL OF MEDICINE (2004)

Article Genetics & Heredity

Insulin receptor splicing alteration in myotonic dystrophy type 2

RS Savkur et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2004)

Review Cell Biology

Regulation of muscle mass by myostatin

SJ Lee

ANNUAL REVIEW OF CELL AND DEVELOPMENTAL BIOLOGY (2004)

Article Multidisciplinary Sciences

A muscleblind knockout model for myotonic dystrophy

RN Kanadia et al.

SCIENCE (2003)

Article Biotechnology & Applied Microbiology

Hammerhead ribozyme-mediated destruction of nuclear foci in myotonic dystrophy myoblasts

MA Langlois et al.

MOLECULAR THERAPY (2003)

Article Biochemistry & Molecular Biology

Viral vector producing antisense RNA restores myotonic dystrophy myoblast functions

D Furling et al.

GENE THERAPY (2003)

Article Clinical Neurology

Creatine monohydrate in DM2/PROMM - A double-blind placebo-controlled clinical study

C Schneider-Gold et al.

NEUROLOGY (2003)

Article Clinical Neurology

Loss of myostatin attenuates severity of muscular dystrophy in mdx mice

KR Wagner et al.

ANNALS OF NEUROLOGY (2002)

Article Multidisciplinary Sciences

Functional improvement of dystrophic muscle by myostatin blockade

S Bogdanovich et al.

NATURE (2002)

Article Multidisciplinary Sciences

Myotonic dystrophy type 2 caused by a CCTG expansion in intron 1 of ZNF9

CL Liquori et al.

SCIENCE (2001)

Article Biochemistry & Molecular Biology

Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy

JW Miller et al.

EMBO JOURNAL (2000)