Cardiac defects contribute to the pathology of spinal muscular atrophy models

Monir Shababi, Javad Habibi, H. T. Yang, +3

Human Molecular Genetics

Cardiac MuscleHeart FailureGeneticsPathologyDisease Gene Identification +23

Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice

Jacqueline Glascock, Erkan Y. Osman, Tristan H. Coady, +3

Journal of Visualized Experiments

A Cytosolic Domain of the Yeast Zrt1 Zinc Transporter Is Required for Its Post-translational Inactivation in Response to Zinc and Cadmium

Raad S. Gitan, Monir Shababi, Michelle D. Kramer, +1

Journal of Biological Chemistry

Molecular BiologyCellular PhysiologyYeastPost-translational InactivationBiological Inorganic Chemistry +15

Development of a Single Vector System that Enhances Trans-Splicing of SMN2 Transcripts

Tristan H. Coady, Travis D. Baughan, Monir Shababi, +2

PLoS ONE

EngineeringGeneticsRna SplicingMolecular BiologyMolecular Genetics +14

Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA Splicing

Tristan H. Coady, Monir Shababi, Gregory E. Tullis, +1

Molecular Therapy

Combination of SMN <i>Trans</i> -Splicing and a Neurotrophic Factor Increases the Life Span and Body Mass in a Severe Model of Spinal Muscular Atrophy

Monir Shababi, Jacqueline Glascock, Christian L. Lorson

Human Gene Therapy

Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector

Travis D. Baughan, Monir Shababi, Tristan H. Coady, +3

Molecular Therapy

Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy

Monir Shababi, Javad Habibi, Lixin Ma, +3

Journal of Molecular and Cellular Cardiology

Severe ModelSpinal Cord InjuryMuscle InjuryPartial RestorationRehabilitation +6

Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy

Jacqueline Glascock, Monir Shababi, Mary J. Wetz, +2

Biochemical and Biophysical Research Communications

Regenerative MedicineSevere ModelNeuroregenerationSpinal Cord InjurySpinal Muscular Atrophy +8

Decreasing Disease Severity in Symptomatic, <i>Smn</i> ^−/− ; <i>SMN2</i> ^+/+ , Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

Jacqueline Glascock, Erkan Y. Osman, Mary J. Wetz, +3

Human Gene Therapy