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

por Rindt, Hansjörg, Tom, Colton M., Lorson, Christian L., Mattis, Virginia B.
Publicado 2017

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 2009

A Direct Comparison of IV and ICV Delivery Methods for Gene Replacement Therapy in a Mouse Model of SMARD1

por Shababi, Monir, Villalón, Eric, Kaifer, Kevin A., DeMarco, Vince, Lorson, Christian L.
Publicado 2018

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 2008

Coronavirus helicases: attractive and unique targets of antiviral drug-development and therapeutic patents

por Spratt, Austin N., Gallazzi, Fabio, Quinn, Thomas P., Lorson, Christian L., Sönnerborg, Anders, Singh, Kamal
Publicado 2021

Mutations in the monkeypox virus replication complex: Potential contributing factors to the 2022 outbreak

por Kannan, Saathvik R., Sachdev, Shrikesh, Reddy, Athreya S., Kandasamy, Shree Lekha, Byrareddy, Siddappa N., Lorson, Christian L., Singh, Kamal
Publicado 2022

Short-duration splice promoting compound enables a tunable mouse model of spinal muscular atrophy

por Rietz, Anne, Hodgetts, Kevin J, Lusic, Hrvoje, Quist, Kevin M, Osman, Erkan Y, Lorson, Christian L, Androphy, Elliot J
Publicado 2020

Survival motor neuron deficiency slows myoblast fusion through reduced myomaker and myomixer expression

por McCormack, Nikki M., Villalón, Eric, Viollet, Coralie, Soltis, Anthony R., Dalgard, Clifton L., Lorson, Christian L., Burnett, Barrington G.
Publicado 2021

Discovery and Evaluation of Entry Inhibitors for SARS-CoV-2 and Its Emerging Variants

por Acharya, Arpan, Pandey, Kabita, Thurman, Michellie, Klug, Elizabeth, Trivedi, Jay, Sharma, Kalicharan, Lorson, Christian L., Singh, Kamal, Byrareddy, Siddappa N.
Publicado 2021

Continued Complexity of Mutations in Omicron Sublineages

por Spratt, Austin N., Kannan, Saathvik R., Sharma, Kalicharan, Sachdev, Shrikesh, Kandasamy, Shree L., Sönnerborg, Anders, Lorson, Christian L., Singh, Kamal
Publicado 2022

Infectivity of SARS-CoV-2: there Is Something More than D614G?

por Kannan, Saathvik R., Spratt, Austin N., Quinn, Thomas P., Heng, Xiao, Lorson, Christian L., Sönnerborg, Anders, Byrareddy, Siddappa N., Singh, Kamal
Publicado 2020

Complex Mutation Pattern of Omicron BA.2: Evading Antibodies without Losing Receptor Interactions

por Kannan, Saathvik R., Spratt, Austin N., Sharma, Kalicharan, Goyal, Ramesh, Sönnerborg, Anders, Apparsundaram, Subbu, Lorson, Christian L., Byrareddy, Siddappa N., Singh, Kamal
Publicado 2022

Comparison of independent screens on differentially vulnerable motor neurons reveals alpha-synuclein as a common modifier in motor neuron diseases

por Kline, Rachel A., Kaifer, Kevin A., Osman, Erkan Y., Carella, Francesco, Tiberi, Ariana, Ross, Jolill, Pennetta, Giuseppa, Lorson, Christian L., Murray, Lyndsay M.
Publicado 2017

Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy

por Comley, Laura H, Kline, Rachel A, Thomson, Alison K, Woschitz, Victoria, Landeros, Eric Villalón, Osman, Erkan Y, Lorson, Christian L, Murray, Lyndsay M
Publicado 2022

Evolution, correlation, structural impact and dynamics of emerging SARS-CoV-2 variants

por Spratt, Austin N., Kannan, Saathvik R., Woods, Lucas T., Weisman, Gary A., Quinn, Thomas P., Lorson, Christian L., Sönnerborg, Anders, Byrareddy, Siddappa N., Singh, Kamal
Publicado 2021

Evolutionary analysis of the Delta and Delta Plus variants of the SARS-CoV-2 viruses

por Kannan, Saathvik R., Spratt, Austin N., Cohen, Alisha R., Naqvi, S. Hasan, Chand, Hitendra S., Quinn, Thomas P., Lorson, Christian L., Byrareddy, Siddappa N., Singh, Kamal
Publicado 2021

Enhancement of SMN protein levels in a mouse model of spinal muscular atrophy using novel drug-like compounds

por Cherry, Jonathan J, Osman, Erkan Y, Evans, Matthew C, Choi, Sungwoon, Xing, Xuechao, Cuny, Gregory D, Glicksman, Marcie A, Lorson, Christian L, Androphy, Elliot J
Publicado 2013

Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2(nmd-2J)

por Shababi, Monir, Smith, Caley E., Ricardez Hernandez, Sara M., Marquez, Jose, Al Rawi, Zayd, Villalón, Eric, Farris, K. David, Garro-Kacher, Mona O., Lorson, Christian L.
Publicado 2021

Functional characterization of SMN evolution in mouse models of SMA

por Osman, Erkan Y., Bolding, Madeline R., Villalón, Eric, Kaifer, Kevin A., Lorson, Zachary C., Tisdale, Sarah, Hao, Yue, Conant, Gavin C., Pires, J. Chris, Pellizzoni, Livio, Lorson, Christian L.
Publicado 2019

ABT1 modifies SMARD1 pathology via interactions with IGHMBP2 and stimulation of ATPase and helicase activity

por Vadla, Gangadhar P., Ricardez Hernandez, Sara M., Mao, Jiude, Garro-Kacher, Mona O., Lorson, Zachary C., Rice, Ronin P., Hansen, Sarah A., Lorson, Christian L., Singh, Kamal, Lorson, Monique A.
Publicado 2023