4.8 Article

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model

Related references

Note: Only part of the references are listed.
Article Biochemistry & Molecular Biology

Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice

Elisa Dominguez et al.

HUMAN MOLECULAR GENETICS (2011)

Article Biochemistry & Molecular Biology

Increased IGF-1 in muscle modulates the phenotype of severe SMA mice

Marta Bosch-Marce et al.

HUMAN MOLECULAR GENETICS (2011)

Article Cell Biology

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

Marco A. Passini et al.

SCIENCE TRANSLATIONAL MEDICINE (2011)

Article Biochemistry & Molecular Biology

Molecular and phenotypic reassessment of an infrequently used mouse model for spinal muscular atrophy

Rocky G. Gogliotti et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2010)

Review Clinical Neurology

Spinal Muscular Atrophy: New and Emerging Insights from Model Mice

Gyu-Hwan Park et al.

CURRENT NEUROLOGY AND NEUROSCIENCE REPORTS (2010)

Article Biochemistry & Molecular Biology

SAHA ameliorates the SMA phenotype in two mouse models for spinal muscular atrophy

Markus Riessland et al.

HUMAN MOLECULAR GENETICS (2010)

Article Biochemistry & Molecular Biology

Arrhythmia and cardiac defects are a feature of spinal muscular atrophy model mice

Christopher R. Heier et al.

HUMAN MOLECULAR GENETICS (2010)

Article Biochemistry & Molecular Biology

Cardiac defects contribute to the pathology of spinal muscular atrophy models

Monir Shababi et al.

HUMAN MOLECULAR GENETICS (2010)

Article Biochemistry & Molecular Biology

Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

Adam K. Bevan et al.

HUMAN MOLECULAR GENETICS (2010)

Review Biochemistry & Molecular Biology

Spinal muscular atrophy: mechanisms and therapeutic strategies

Christian L. Lorson et al.

HUMAN MOLECULAR GENETICS (2010)

Article Medicine, Research & Experimental

CNS-targeted gene therapy improves survival and motor function in a mouse model of spinal muscular atrophy

Marco A. Passini et al.

JOURNAL OF CLINICAL INVESTIGATION (2010)

Article Biotechnology & Applied Microbiology

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Kevin D. Foust et al.

NATURE BIOTECHNOLOGY (2010)

Review Endocrinology & Metabolism

Human Acid-Labile Subunit Deficiency: Clinical, Endocrine and Metabolic Consequences

Horacio M. Domene et al.

HORMONE RESEARCH (2009)

Review Neurosciences

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

Arthur H. M. Burghes et al.

NATURE REVIEWS NEUROSCIENCE (2009)

Article Genetics & Heredity

Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in Transgenic mice

Yimin Hua et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2008)

Review Endocrinology & Metabolism

The GH-IGF-I axis and the cardiovascular system: clinical implications

Annamaria Colao

CLINICAL ENDOCRINOLOGY (2008)

Article Clinical Neurology

Subcutaneous IGF-1 is not beneficial in 2-year ALS trial

E. J. Sorenson et al.

NEUROLOGY (2008)

Article Genetics & Heredity

Congenital heart disease is a feature of severe infantile spinal muscular atrophy

S. Rudnik-Schoeneborn et al.

JOURNAL OF MEDICAL GENETICS (2008)

Article Multidisciplinary Sciences

The power of automated high-resolution behavior analysis revealed by its application to mouse models of Huntington's and prion diseases

Andrew D. Steele et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)

Review Biochemistry & Molecular Biology

The plasticity of aging: Insights from long-lived mutants

C Kenyon

Review Genetics & Heredity

Role of insulin-like growth factor I signaling in neurodegenerative diseases

JL Trejo et al.

JOURNAL OF MOLECULAR MEDICINE-JMM (2004)

Article Multidisciplinary Sciences

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model

BK Kaspar et al.

SCIENCE (2003)