Monir Shababi

A cytosolic domain of the yeast Zrt1 zinc transporter is required for its post-translational inactivation in response to zinc and cadmium.

The ribosomal shunt translation strategy of cauliflower mosaic virus has evolved from ancient long terminal repeats.

Stimulating full-length SMN2 expression by delivering bifunctional RNAs via a viral vector.

Restoration of SMN function: delivery of a trans-splicing RNA re-directs SMN2 pre-mRNA splicing.

Development of a single vector system that enhances trans-splicing of SMN2 transcripts.

Spinal muscular atrophy: mechanisms and therapeutic strategies.

Cardiac defects contribute to the pathology of spinal muscular atrophy models.

Combination of SMN trans-splicing and a neurotrophic factor increases the life span and body mass in a severe model of spinal muscular atrophy.

Therapeutics that directly increase SMN expression to treat spinal muscular atrophy.

Optimization of SMN Trans-Splicing Through the Analysis of SMN Introns.

Decreasing Disease Severity in Symptomatic, Smn(-/-);SMN2(+/+), Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery.

Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy.

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