Trans-Splicing-Mediated Improvement in a Severe Mouse Model of Spinal Muscular Atrophy

Por Coady, Tristan H., Lorson, Christian L.
Publicado em 2010

SMN-inducing compounds for the treatment of spinal muscular atrophy

Por Lorson, Monique A, Lorson, Christian L
Publicado em 2012

Spinal muscular atrophy: mechanisms and therapeutic strategies

Por Lorson, Christian L., Rindt, Hansjorg, Shababi, Monir
Publicado em 2010

A Negatively Acting Bifunctional RNA Increases Survival Motor Neuron Both In Vitro and In Vivo

Por Dickson, Alexa, Osman, Erkan, Lorson, Christian L.
Publicado em 2008

Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?

Por Shababi, Monir, Lorson, Christian L, Rudnik-Schöneborn, Sabine S
Publicado em 2014

A SMNΔ7 read-through product confers functionality to the SMNΔ7 protein

Por Mattis, Virginia, Bowman, Melissa, Kothary, Rashmi, Lorson, Christian L.
Publicado em 2008

Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy

Por Osman, Erkan Y, Yen, Pei-Fen, Lorson, Christian L
Publicado em 2012

A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy

Por Lorson, Christian L., Hahnen, Eric, Androphy, Elliot J., Wirth, Brunhilde
Publicado em 1999

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

Por Mattis, Virginia B., Butchbach, Matthew E. R., Lorson, Christian L.
Publicado em 2008

Analysis of a read-through promoting compound in a severe mouse model of Spinal Muscular Atrophy

Por Mattis, Virginia B., Chang, Cheng-Wei Tom, Lorson, Christian L.
Publicado em 2012

The identification and characterization of the porcine (Sus scrofa) Survival Motor Neuron (SMN1) gene: An animal model for therapeutic studies

Por Lorson, Monique A., Spate, Lee D., Prather, Randall S., Lorson, Christian L.
Publicado em 2008

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

Por Rose, Ferrill F., Mattis, Virginia B., Rindt, Hansjörg, Lorson, Christian L.
Publicado em 2009

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

Por Baughan, Travis D., Dickson, Alexa, Osman, Erkan Y., Lorson, Christian L.
Publicado em 2009

Optimization of trans-Splicing for Huntington's Disease RNA Therapy

Por Rindt, Hansjörg, Tom, Colton M., Lorson, Christian L., Mattis, Virginia B.
Publicado no Front Neurosci (2017)

Htra2-β1 stimulates an exonic splicing enhancer and can restore full-length SMN expression to survival motor neuron 2 (SMN2)

Por Hofmann, Yvonne, Lorson, Christian L., Stamm, Stefan, Androphy, Elliot J., Wirth, Brunhilde
Publicado em 2000

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

Por Mattis, Virginia B, Fosso, Marina Y, Chang, Cheng-Wei, Lorson, Christian L
Publicado em 2009

Restoration of SMN to Emx-1 expressing cortical neurons is not sufficient to provide benefit to a severe mouse model of Spinal Muscular Atrophy

Por Taylor, Alexander S., Glascock, Jacqueline J., Rose, Ferrill F., Lutz, Cathleen, Lorson, Christian L.
Publicado em 2013

Minute Virus of Mice NS1 Interacts with the SMN Protein, and They Colocalize in Novel Nuclear Bodies Induced by Parvovirus Infection

Por Young, Philip J., Jensen, Klaus T., Burger, Lisa R., Pintel, David J., Lorson, Christian L.
Publicado em 2002

Minute Virus of Mice Small Nonstructural Protein NS2 Interacts and Colocalizes with the Smn Protein

Por Young, Philip J., Jensen, Klaus T., Burger, Lisa R., Pintel, David J., Lorson, Christian L.
Publicado em 2002

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

Por Coady, Tristan H., Baughan, Travis D., Shababi, Monir, Passini, Marco A., Lorson, Christian L.
Publicado em 2008