Works by Lorson, Christian

Results: 66

The Nefl<sup>E397K</sup> mouse model demonstrates muscle pathology and motor function deficits consistent with CMT2E.

Published in:

Human Molecular Genetics, 2025, v. 34, n. 15, p. 1313, doi. 10.1093/hmg/ddaf080

By:

Pérez-López, Dennis O;
Shively, Audrey A;
Torres, F Javier Llorente;
Muchow, Roxanne;
Abu-Salah, Zaid;
Abu-Salah, Mohammed T;
Veltrop, Jackson T;
Garcia, Michael L;
Smith, Catherine L;
Cornelison, D D W;
Nichols, Nicole L;
Lorson, Monique A;
Lorson, Christian

Publication type:

Article

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

Published in:

Human Molecular Genetics, 2022, v. 31, n. 18, p. 3107, doi. 10.1093/hmg/ddac097

By:

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

Publication type:

Article

combinatorial approach increases SMN level in SMA model mice.

Published in:

Human Molecular Genetics, 2022, v. 31, n. 17, p. 2989, doi. 10.1093/hmg/ddac068

By:

Dumas, Samantha A;
Villalón, Eric;
Bergman, Elizabeth M;
Wilson, Kenneth J;
Marugan, Juan J;
Lorson, Christian L;
Burnett, Barrington G

Publication type:

Article

Ighmbp2D564N mouse model is the first SMARD1 model to demonstrate respiratory defects.

Published in:

Human Molecular Genetics, 2022, v. 31, n. 8, p. 1293, doi. 10.1093/hmg/ddab317

By:

Smith, Caley E;
Lorson, Monique A;
Hernandez, Sara M Ricardez;
Rawi, Zayd Al;
Mao, Jiude;
Marquez, Jose;
Villalón, Eric;
Keilholz, Amy N;
Smith, Catherine L;
Garro-Kacher, Mona O;
Morcos, Toni;
Davis, Daniel J;
Bryda, Elizabeth C;
Nichols, Nicole L;
Lorson, Christian L

Publication type:

Article

adeno-associated virus 2 genome and Rep 68/78 proteins interact with cellular sites of DNA damage.

Published in:

Human Molecular Genetics, 2022, v. 31, n. 6, p. 985, doi. 10.1093/hmg/ddab300

By:

Boftsi, Maria;
Whittle, Fawn B;
Wang, Juexin;
Shepherd, Phoenix;
Burger, Lisa R;
Kaifer, Kevin A;
Lorson, Christian L;
Joshi, Trupti;
Pintel, David J;
Majumder, Kinjal

Publication type:

Article

Islatravir: evaluation of clinical development for HIV and HBV.

Published in:

2024

By:

Gillespie, Samuel W.;
Reddy, Athreya S.;
Burris, Dana M.;
Naqvi, S. Hasan;
Byrareddy, Siddappa N.;
Lorson, Christian L.;
Singh, Kamal

Publication type:

Report

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

Published in:

PLoS Genetics, 2017, v. 13, n. 3, p. 1, doi. 10.1371/journal.pgen.1006680

By:

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

Publication type:

Article

CRISPR-Mediated Enzyme Fragment Complementation Assay for Quantification of the Stability of Splice Isoforms.

Published in:

ChemBioChem, 2022, v. 23, n. 9, p. 1, doi. 10.1002/cbic.202200012

By:

Zhichao Tang;
Hegde, Shalakha;
Junxing Zhao;
Shoutian Zhu;
Johnson, Kristen A.;
Lorson, Christian L.;
Jingxin Wang

Publication type:

Article

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

Published in:

Journal of Anatomy, 2014, v. 224, n. 1, p. 15, doi. 10.1111/joa.12083

By:

Shababi, Monir;
Lorson, Christian L.;
Rudnik-Schöneborn, Sabine S.

Publication type:

Article

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

Published in:

Journal of Neuroscience, 2010, v. 30, n. 1, p. 126, doi. 10.1523/JNEUROSCI.4489-09.2010

By:

Coady, Tristan H.;
Lorson, Christian L.

Publication type:

Article

Survival Motor Neuron Function in Motor Axons Is Independent of Functions Required for Small Nuclear Ribonucleoprotein Biogenesis.

Published in:

Journal of Neuroscience, 2006, v. 26, n. 43, p. 11014, doi. 10.1523/JNEUROSCI.1637-06.2006

By:

Carrel, Tessa L.;
McWhorter, Michelle L.;
Workman, Eileen;
Honglai Zhang;
Wolstencroft, Elizabeth C.;
Lorson, Christian;
Bassell, Gary J.;
Burghes, Arthur H. M.;
Beattie, Christine E.

Publication type:

Article

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

Published in:

Journal of Molecular Neuroscience, 2012, v. 46, n. 3, p. 459, doi. 10.1007/s12031-011-9614-3

By:

Shababi, Monir;
Lorson, Christian

Publication type:

Article

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

Published in:

Developmental Dynamics, 2008, v. 237, n. 8, p. 2268, doi. 10.1002/dvdy.21642

By:

Lorson, Monique A.;
Spate, Lee D.;
Prather, Randall S.;
Lorson, Christian L.

Publication type:

Article

Induced pluripotent stem cells from a spinal muscular atrophy patient.

Published in:

Nature, 2009, v. 457, n. 7227, p. 277, doi. 10.1038/nature07677

By:

Ebert, Allison D.;
Yu, Junying;
Rose, Ferrill F.;
Mattis, Virginia B.;
Lorson, Christian L.;
Thomson, James A.;
Svendsen, Clive N.

Publication type:

Article

Trans cohort metabolic reprogramming towards glutaminolysis in long-term successfully treated HIV-infection.

Published in:

Communications Biology, 2022, v. 5, n. 1, p. 1, doi. 10.1038/s42003-021-02985-3

By:

Mikaeloff, Flora;
Svensson Akusjärvi, Sara;
Ikomey, George Mondinde;
Krishnan, Shuba;
Sperk, Maike;
Gupta, Soham;
Magdaleno, Gustavo Daniel Vega;
Escós, Alejandra;
Lyonga, Emilia;
Okomo, Marie Claire;
Tagne, Claude Tayou;
Babu, Hemalatha;
Lorson, Christian L.;
Végvári, Ákos;
Banerjea, Akhil C.;
Kele, Julianna;
Hanna, Luke Elizabeth;
Singh, Kamal;
de Magalhães, João Pedro;
Benfeitas, Rui

Publication type:

Article

Inhibitors against DNA Polymerase I Family of Enzymes: Novel Targets and Opportunities.

Published in:

Biology (2079-7737), 2024, v. 13, n. 4, p. 204, doi. 10.3390/biology13040204

By:

Kannan, Saathvik;
Gillespie, Samuel W.;
Picking, Wendy L.;
Picking, William D.;
Lorson, Christian L.;
Singh, Kamal

Publication type:

Article

Correction: Applications of Surface Plasmon Resonance (SPR) to the Study of Diverse Protein‐Ligand Interactions.

Published in:

Current Protocols, 2024, v. 4, n. 7, p. 1, doi. 10.1002/cpz1.1108

By:

Burris, Dana M.;
Gillespie, Samuel W.;
Campbell, Emma Joy;
Ice, S. Nick;
Yadav, Vikas;
Picking, William D.;
Lorson, Christian L.;
Singh, Kamal

Publication type:

Article

Applications of Surface Plasmon Resonance (SPR) to the Study of Diverse Protein‐Ligand Interactions.

Published in:

Current Protocols, 2024, v. 4, n. 6, p. 1, doi. 10.1002/cpz1.1030

By:

Burris, Dana M.;
Gillespie, Samuel W.;
Campbell, Emma Joy;
Ice, S. Nick;
Yadav, Vikas;
Picking, William D.;
Lorson, Christian L.;
Singh, Kamal

Publication type:

Article

AAV9-mediated delivery of miR-23a reduces disease severity in Smn<sup>2B/−</sup>SMA model mice.

Published in:

Human Molecular Genetics, 2019, v. 28, n. 19, p. 3199, doi. 10.1093/hmg/ddz142

By:

Kaifer, Kevin A;
Villalón, Eric;
O'Brien, Benjamin S;
Sison, Samantha L;
Smith, Caley E;
Simon, Madeline E;
Marquez, Jose;
O'Day, Siri;
Hopkins, Abigail E;
Neff, Rachel;
Rindt, Hansjörg;
Ebert, Allison D;
Lorson, Christian L

Publication type:

Article

Selective vulnerability in neuronal populations in nmd/SMARD1 mice.

Published in:

Human Molecular Genetics, 2018, v. 27, n. 4, p. 679, doi. 10.1093/hmg/ddx434

By:

Villalón, Eric;
Shababi, Monir;
Kline, Rachel;
Lorson, Zachary C.;
Florea, Kyra M.;
Lorson, Christian L.

Publication type:

Article

Astrocyte-produced miR-146a as a mediator of motor neuron loss in spinal muscular atrophy.

Published in:

Human Molecular Genetics, 2017, v. 26, n. 17, p. 3409, doi. 10.1093/hmg/ddx230

By:

Sison, Samantha L.;
Patitucci, Teresa N.;
Seminary, Emily R.;
Villalon, Eric;
Lorson, Christian L.;
Ebert, Allison D.

Publication type:

Article

SMN deficiency negatively impacts red pulp macrophages and spleen development in mouse models of spinal muscular atrophy.

Published in:

Human Molecular Genetics, 2017, v. 26, n. 5, p. 932, doi. 10.1093/hmg/ddx008

By:

Khairallah, Marie-Therese;
Astroski, Jacob;
Custer, Sarah K.;
Androphy, Elliot J.;
Franklin, Craig L.;
Lorson, Christian L.

Publication type:

Article

Astrocytes influence the severity of spinal muscular atrophy.

Published in:

Human Molecular Genetics, 2015, v. 24, n. 14, p. 4094, doi. 10.1093/hmg/ddv148

By:

Rindt, Hansjörg;
Zhihua Feng;
Mazzasette, Chiara;
Glascock, Jacqueline J.;
Valdivia, David;
Pyles, Noah;
Crawford, Thomas O.;
Swoboda, Kathryn J.;
Patitucci, Teresa N.;
Ebert, Allison D.;
Sumner, Charlotte J.;
Ko, Chien-Ping;
Lorson, Christian L.

Publication type:

Article

The SMN structure reveals its crucial role in snRNP assembly.

Published in:

Human Molecular Genetics, 2015, v. 24, n. 8, p. 2138, doi. 10.1093/hmg/ddu734

By:

Seng, Chenda O.;
Magee, Craig;
Young, Philip J.;
Lorson, Christian L.;
Allen, James P.

Publication type:

Article

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models.

Published in:

Human Molecular Genetics, 2014, v. 23, n. 18, p. 4832, doi. 10.1093/hmg/ddu198

By:

Osman, Erkan Y.;
Miller, Madeline R.;
Robbins, Kate L.;
Lombardi, Abby M.;
Atkinson, Arleigh K.;
Brehm, Amanda J.;
Lorson, Christian L.

Publication type:

Article

Defining the therapeutic window in a severe animal model of spinal muscular atrophy.

Published in:

Human Molecular Genetics, 2014, v. 23, n. 17, p. 4559, doi. 10.1093/hmg/ddu169

By:

Robbins, Kate L.;
Glascock, Jacqueline J.;
Osman, Erkan Y.;
Miller, Madeline R.;
Lorson, Christian L.

Publication type:

Article

Development and characterization of an SMN2-based intermediate mouse model of Spinal Muscular Atrophy.

Published in:

Human Molecular Genetics, 2013, v. 22, n. 9, p. 1843, doi. 10.1093/hmg/ddt037

By:

Cobb, Melissa S.;
Rose, Ferril F.;
Rindt, Hansjörg;
Glascock, Jacqueline J.;
Shababi, Monir;
Miller, Madeline R.;
Osman, Erkan Y.;
Yen, Pei-Fen;
Garcia, Michael L.;
Martin, Brittanie R.;
Wetz, Mary J.;
Mazzasette, Chiara;
Feng, Zhihua;
Ko, Chien-Ping;
Lorson, Christian L.

Publication type:

Article

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

Published in:

Human Molecular Genetics, 2011, v. 20, n. 9, p. 1701, doi. 10.1093/hmg/ddr046

By:

Peter, Cyril Jayakumar;
Evans, Matthew;
Thayanithy, Venugopal;
Taniguchi-Ishigaki, Naoko;
Bach, Ingolf;
Kolpak, Adrianne;
Bassell, Gary J.;
Rossoll, Wilfried;
Lorson, Christian L.;
Bao, Zheng-Zheng;
Androphy, Elliot J.

Publication type:

Article

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

Published in:

Human Molecular Genetics, 2010, v. 19, n. 20, p. 4059, doi. 10.1093/hmg/ddq329

By:

Shababi, Monir;
Habibi, Javad;
Yang, Hsiao T.;
Vale, Spencer M.;
Sewell, Will A.;
Lorson, Christian L.

Publication type:

Article

Spinal muscular atrophy: mechanisms and therapeutic strategies.

Published in:

Human Molecular Genetics, 2010, v. 19, n. R1, p. R111, doi. 10.1093/hmg/ddq147

By:

Lorson, Christian L.;
Rindt, Hansjorg;
Shababi, Monir

Publication type:

Article

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

Published in:

Human Molecular Genetics, 2009, v. 18, n. 20, p. 3906, doi. 10.1093/hmg/ddp333

By:

Mattis, Virginia B.;
Ebert, Allison D.;
Fosso, Marina Y.;
Chang, Cheng-Wei;
Lorson, Christian L.

Publication type:

Article

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

Published in:

Human Molecular Genetics, 2009, v. 18, n. 9, p. 1600, doi. 10.1093/hmg/ddp076

By:

Baughan, Travis D.;
Dickson, Alexa;
Osman, Erkan Y.;
Lorson, Christian L.

Publication type:

Article

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

Published in:

Human Molecular Genetics, 2009, v. 18, n. 6, p. 997, doi. 10.1093/hmg/ddn426

By:

Rose, Ferrill F.;
Mattis, Virginia B.;
Rindt, Hansjörg;
Lorson, Christian L.

Publication type:

Article

Replacement of huntingtin exon 1 by trans-splicing.

Published in:

Cellular & Molecular Life Sciences, 2012, v. 69, n. 24, p. 4191, doi. 10.1007/s00018-012-1083-5

By:

Rindt, Hansjörg;
Yen, Pei-Fen;
Thebeau, Christina;
Peterson, Troy;
Weisman, Gary;
Lorson, Christian

Publication type:

Article

Red Cabbage Juice-Mediated Gut Microbiota Modulation Improves Intestinal Epithelial Homeostasis and Ameliorates Colitis.

Published in:

International Journal of Molecular Sciences, 2024, v. 25, n. 1, p. 539, doi. 10.3390/ijms25010539

By:

Jean Wilson, Emily;
Sirpu Natesh, Nagabhishek;
Ghadermazi, Parsa;
Pothuraju, Ramesh;
Prajapati, Dipakkumar R.;
Pandey, Sanjit;
Kaifi, Jussuf T.;
Dodam, John R.;
Bryan, Jeffrey N.;
Lorson, Christian L.;
Watrelot, Aude A.;
Foster, Jason M.;
Mansell, Thomas J.;
Joshua Chan, Siu Hung;
Batra, Surinder K.;
Subbiah, Jeyamkondan;
Rachagani, Satyanarayana

Publication type:

Article

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

Published in:

International Journal of Molecular Sciences, 2022, v. 23, n. 10, p. 5534, doi. 10.3390/ijms23105534

By:

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

Publication type:

Article

Continued Complexity of Mutations in Omicron Sublineages.

Published in:

Biomedicines, 2022, v. 10, n. 10, p. N.PAG, doi. 10.3390/biomedicines10102593

By:

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

Publication type:

Article

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

Published in:

Journal of Cachexia, Sarcopenia & Muscle, 2021, v. 12, n. 4, p. 1098, doi. 10.1002/jcsm.12740

By:

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

Publication type:

Article

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

Published in:

Frontiers in Neuroscience, 2017, p. 1, doi. 10.3389/fnins.2017.00544

By:

Rindt, Hansjörg;
Tom, Colton M.;
Lorson, Christian L.;
Mattis, Virginia B.

Publication type:

Article

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

Published in:

2020

By:

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

Publication type:

Letter

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

Published in:

Acta Neuropathologica, 2011, v. 122, n. 3, p. 331, doi. 10.1007/s00401-011-0848-5

By:

Dale, Jeffrey;
Shen, Hailian;
Barry, Devin;
Garcia, Virginia;
Rose, Ferrill;
Lorson, Christian;
Garcia, Michael

Publication type:

Article

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

Published in:

Transgenic Research, 2013, v. 22, n. 5, p. 1029, doi. 10.1007/s11248-013-9702-y

By:

Taylor, Alexander;
Glascock, Jacqueline;
Rose, Ferrill;
Lutz, Cathleen;
Lorson, Christian

Publication type:

Article

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

Published in:

Transgenic Research, 2011, v. 20, n. 6, p. 1293, doi. 10.1007/s11248-011-9496-8

By:

Lorson, Monique;
Spate, Lee;
Samuel, Melissa;
Murphy, Clifton;
Lorson, Christian;
Prather, Randall;
Wells, Kevin

Publication type:

Article

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

Published in:

Molecular Therapy, 2012, v. 20, n. 1, p. 119, doi. 10.1038/mt.2011.232

By:

Osman, Erkan Y;
Yen, Pei-Fen;
Lorson, Christian L

Publication type:

Article

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

Published in:

Molecular Therapy, 2007, v. 15, n. 8, p. 1471, doi. 10.1038/sj.mt.6300222

By:

Coady, Tristan H.;
Shababi, Monir;
Tullis, Gregory E.;
Lorson, Christian L.

Publication type:

Article

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

Published in:

Molecular Therapy, 2006, v. 14, n. 1, p. 54, doi. 10.1016/j.ymthe.2006.01.012

By:

Baughan, Travis;
Shababi, Monir;
Coady, Tristan H.;
Dickson, Alexa M.;
Tullis, Gregory E.;
Lorson, Christian L.

Publication type:

Article

253. AAV Delivery of a Trans-Splicing RNA Re-Directs SMN2 Splicing and Results in Increased Full-Length SMN.

Published in:

Molecular Therapy, 2006, v. 13, p. S97, doi. 10.1016/j.ymthe.2006.08.280

By:

H. Coady, Tristan;
Shababi, Monir;
Lorson, Christian L.

Publication type:

Article

415. Stimulating Full-Length SMN2 Expression by Delivering Bi-Functional RNAs Via a Viral Vector.

Published in:

Molecular Therapy, 2006, v. 13, p. S159, doi. 10.1016/j.ymthe.2006.08.479

By:

Baughan, Travis D.;
Shababi, Monir;
Tullis, Gregory E.;
Lorson, Christian L.

Publication type:

Article

Functional characterization of SMN evolution in mouse models of SMA.

Published in:

Scientific Reports, 2019, v. 9, n. 1, p. N.PAG, doi. 10.1038/s41598-019-45822-8

By:

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.

Publication type:

Article

Potential therapeutic targets for Mpox: the evidence to date.

Published in:

2023

By:

Byrareddy, Siddappa N;
Sharma, Kalicharan;
Sachdev, Shrikesh;
Reddy, Athreya S.;
Acharya, Arpan;
Klaustermeier, Kaylee M.;
Lorson, Christian L;
Singh, Kamal

Publication type:

Report