Christian L. Lorson

A direct interaction between the survival motor neuron protein and p53 and its relationship to spinal muscular atrophy.

SRp30c-dependent stimulation of survival motor neuron (SMN) exon 7 inclusion is facilitated by a direct interaction with hTra2 beta 1.

Minute virus of mice NS1 interacts with the SMN protein, and they colocalize in novel nuclear bodies induced by parvovirus infection.

Minute virus of mice small nonstructural protein NS2 interacts and colocalizes with the Smn protein.

Survival motor neuron protein in the nucleolus of mammalian neurons.

The Ewing's sarcoma protein interacts with the Tudor domain of the survival motor neuron protein.

A survival motor neuron:tetanus toxin fragment C fusion protein for the targeted delivery of SMN protein to neurons.

Monitoring of recombinant survival motor neuron protein using fiber-optic surface plasmon resonance.

Minute virus of mice small non-structural protein NS2 localizes within, but is not required for the formation of, Smn-associated autonomous parvovirus-associated replication bodies.

A non-sequence-specific requirement for SMN protein activity: the role of aminoglycosides in inducing elevated SMN protein levels.

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

Novel aminoglycosides increase SMN levels in spinal muscular atrophy fibroblasts.

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

Protein phosphatase 1 binds to the RNA recognition motif of several splicing factors and regulates alternative pre-mRNA processing.

Induction of full-length survival motor neuron by polyphenol botanical compounds.

A SMNDelta7 read-through product confers functionality to the SMNDelta7 protein.

Identification and characterization of the porcine (Sus scrofa) survival motor neuron (SMN1) gene: an animal model for therapeutic studies.

Identification and characterisation of a nuclear localisation signal in the SMN associated protein, Gemin4.

The Wallerian degeneration slow (Wld(s)) gene does not attenuate disease in a mouse model of spinal muscular atrophy.

Detection of human survival motor neuron (SMN) protein in mice containing the SMN2 transgene: applicability to preclinical therapy development for spinal muscular atrophy.

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

Delivery of recombinant follistatin lessens disease severity in a mouse model of spinal muscular atrophy.

Induced pluripotent stem cells from a spinal muscular atrophy patient.

Delivery of bifunctional RNAs that target an intronic repressor and increase SMN levels in an animal model of spinal muscular atrophy.

Delivery of a read-through inducing compound, TC007, lessens the severity of a spinal muscular atrophy animal model.

A negatively acting bifunctional RNA increases survival motor neuron both in vitro and in vivo.

Identification of a tripartite import signal in the Ewing Sarcoma protein (EWS).

RNA-targeting approaches for neuromuscular diseases.

Effect of diet on the survival and phenotype of a mouse model for spinal muscular atrophy.

Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA.

Trans-splicing-mediated improvement in a severe mouse model of spinal muscular atrophy.

Identification of a self-association domain in the Ewing's sarcoma protein: a novel function for arginine-glycine-glycine rich motifs?

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.

The COPI vesicle complex binds and moves with survival motor neuron within axons.

Disruption of the Survival Motor Neuron (SMN) gene in pigs using ssDNA.

The spinal muscular atrophy mouse model, SMAΔ7, displays altered axonal transport without global neurofilament alterations.

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

SMN in spinal muscular atrophy and snRNP biogenesis.

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

Bifunctional RNAs targeting the intronic splicing silencer N1 increase SMN levels and reduce disease severity in an animal model of spinal muscular atrophy.

Transgenic inactivation of murine myostatin does not decrease the severity of disease in a model of Spinal Muscular Atrophy.

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.