RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Cited by (687)

Items 101–200 of 687

  1. Astrocytes influence the severity of spinal muscular atrophy
    Hansjörg Rindt, Zhihua Feng, Chiara Mazzasette, Jacqueline Glascock, David O. Valdivia, Noah J. Pyles, Thomas O. Crawford, Kathryn J. Swoboda, Teresa N. Patitucci, Allison D. Ebert et al. · 2015 · Human Molecular Genetics
  2. A Novel Morpholino Oligomer Targeting ISS-N1 Improves Rescue of Severe Spinal Muscular Atrophy Transgenic Mice
    Haiyan Zhou, Narinder Janghra, Chalermchai Mitrpant, Rachel L. Dickinson, Karen Anthony, Loren L. Flynn, Ian C. Eperon, Steve Donald Wilton, Jennifer Elizabeth Morgan, Francesco Muntoni A · 2013 · Human Gene Therapy
  3. Viral Vectors and Delivery Strategies for CNS Gene Therapy
    Steven James Gray, Kenton Woodard, Richard Jude Samulski · 2010 · Therapeutic Delivery
  4. Brain microvasculature defects and Glut1 deficiency syndrome averted by early repletion of the glucose transporter-1 protein
    Maoxue Tang, Guangping Gao, Carlos B. Rueda, Hang Yu, David N. Thibodeaux, Tomoyuki Awano, Kristin Engelstad, Maria J. Sanchez‐Quintero, Hong Yang, Fanghua Li et al. · 2017 · Nature Communications
  5. Therapeutic strategies for spinal muscular atrophy: SMN and beyond
    Mélissa Bowerman, Catherina Gwynne Becker, Rafael J. Yáñez‐Muñoz, Ke Ning, Matthew J. A. Wood, Thomas Henry Gillingwater, Kevin Talbot · 2017 · Disease Models & Mechanisms
  6. Adeno-Associated Viral Vectors in Neuroscience Research
    David L. Haggerty, Gregory G. Grecco, Kaitlin C. Reeves, Brady K. Atwood · 2019 · Molecular Therapy — Methods & Clinical Development
  7. Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
    Karim Bey, Johan Deniaud, Laurence Dubreil, Béatrice Joussemet, Joseph Cristini, Carine Ciron, Juliette Hordeaux, Morwenn Le Boulc’h, Kévin Marche, Maud Maquigneau et al. · 2020 · Molecular Therapy — Methods & Clinical Development
  8. Genetically modified pig models for neurodegenerative disorders
    Ida E. Holm, Aage Kristian Olsen Alstrup, Yonglun Luo · 2015 · The Journal of Pathology
  9. Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases
    Perez, Barbara A., Shutterly, Alison, Chan, Ying Kai, Byrne, Barry J., Corti, Manuela · 2020 · Brain Sciences
  10. In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy
    Sourav Choudhury, Zachary Fitzpatrick, Anne F Harris, Stacy A. Maitland, Jennifer S Ferreira, Yuanfan Zhang, Shan Ma, Rohit B. Sharma, Heather L. Gray‐Edwards, Jacob A. Johnson et al. · 2016 · Molecular Therapy
  11. Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector
    Sourav Choudhury, Anne F Harris, Damien J. Cabral, Allison M. Keeler, Ellen Sapp, Jennifer S Ferreira, Heather L. Gray‐Edwards, Jacob A. Johnson, Aime K. Johnson, Qin Su et al. · 2015 · Molecular Therapy
  12. Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1 G93A Mice and Nonhuman Primates
    Florie Borel, Gwladys Gernoux, Brynn H. Cardozo, Jake Metterville, Gabriela Toro Cabrera, Lina Song, Qin Su, Guang Ping Gao, Mai K. ElMallah, Robert H. Brown et al. · 2015 · Human Gene Therapy
  13. Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection
    Jesse R. McLean, Gaynor Ann Smith, Emily M. Rocha, Melissa A. Hayes, Jonathan A. Beagan, Penelope J. Hallett, Ole Isacson A · 2014 · Neuroscience Letters
  14. Methods for Gene Transfer to the Central Nervous System
    Kantor, Boris, Bailey, Rachel M., Wimberly, Keon, Kalburgi, Sahana N., Gray, Steven J. · 2014 · Advances in Genetics
  15. The secretome of periodontal ligament stem cells from MS patients protects against EAE
    Rajan, Thangavelu Soundara, Giacoppo, Sabrina, Diomede, Francesca, Ballerini, Patrizia, Paolantonio, Michele, Marchisio, Marco, Piattelli, Adriano, Bramanti, Placido, Mazzon, Emanuela, Trubiani, Oriana · 2016 · Scientific Reports
  16. Novel targeted therapies for Parkinson’s disease
    Theodora Ntetsika, Paraskevi-Evita Papathoma, Ioanna Markaki · 2021 · Molecular Medicine
  17. Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease
    Mohammad A. Rafi, Han Zhi Rao, Paola Luzi, Mark T. Curtis, David A. Wenger · 2012 · Molecular Therapy
  18. Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease
    Valentina Sardone, Haiyan Zhou, Francesco Muntoni, Alessandra Ferlini, Maria Sofia Falzarano · 2017 · Molecules
  19. Muscle-specific SMN reduction reveals motor neuron–independent disease in spinal muscular atrophy models
    Jeong-Ki Kim, Narendra Nath Jha, Zhihua Feng, Michelle R. Faleiro, Claudia A. Chiriboga, Lan Wei‐LaPierre, Robert T. Dirksen, Chien‐Ping Ko, Umrao R. Monani · 2020 · Journal of Clinical Investigation
  20. Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice
    Tanguy, Yannick, Biferi, Maria G., Besse, Aurore, Astord, Stephanie, Cohen-Tannoudji, Mathilde, Marais, Thibaut, Barkats, Martine · 2015 · Frontiers in Molecular Neuroscience
  21. Systemic delivery of adeno-associated viral vectors
    Dongsheng Duan · 2016 · Current Opinion in Virology
  22. Drug treatment for spinal muscular atrophy types II and III
    Renske I. Wadman, Wendy MJ Bosboom, W. Ludo van der Pol, Leonard H. van den Berg, John H. J. Wokke, Susan T. Iannaccone, Alexander F. J. E. Vrancken · 2012 · Cochrane Database of Systematic Reviews
  23. Splicing repression is a major function of TDP-43 in motor neurons
    Aneesh Donde, Mingkuan Sun, Jonathan P. Ling, Kerstin E. Braunstein, Bo Pang, Xinrui Wen, Xueying Cheng, Liam Chen, Philip C. Wong · 2019 · Acta Neuropathologica
  24. Ly6a Differential Expression in Blood–Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B
    Ana Rita Batista, Oliver D. King, Christopher P. Reardon, Crystal Davis, Shankaracharya, Vivek M. Philip, Heather L. Gray‐Edwards, Neil Aronin M. D, Cathleen M. Lutz, John E. Landers et al. · 2019 · Human Gene Therapy
  25. A Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan Mice
    Seemin Seher Ahmed, Huapeng Li, Chunyan Cao, Elif Muazzez Şıkoğlu, Andrew R. Denninger, Qin Su, Samuel C. Eaton, Ana A Liso Navarro, Jun Xie, Sylvia Szucs et al. · 2013 · Molecular Therapy
  26. Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system
    Ahad A. Rahim, Andrew M.S. Wong, Klemens Hoefer, Suzanne M. K. Buckley, Citra Nurfarah Zaini Mattar, Seng H. Cheng, Jerry Kok Yen Chan, Jonathan D. Cooper, Simon N. Waddington · 2011 · The FASEB Journal
  27. Spinal Muscular Atrophy: From Gene Discovery to Clinical Trials
    Dian Kesumapramudya Nurputra, Poh San Lai, Nur Imma Fatimah Harahap, Satoru Morikawa, Tomoto Yamamoto, Noriyuki Nishimura, Yuji Kubo, Atsuko Takeuchi, Toshio Saito, Yasuhiro Takeshima et al. · 2013 · Annals of Human Genetics
  28. Gene Therapy for ALS—A Perspective
    M Cappella, Chiara Ciotti, Mathilde Cohen-Tannoudji, Maria Grazia Biferi · 2019 · International Journal of Molecular Sciences
  29. Gene Therapy for the Nervous System: Challenges and New Strategies
    Casey A. Maguire, Servio H. Ramirez, Steven F. Merkel, Miguel Sena‐Esteves, Xandra O. Breakefield · 2014 · Neurotherapeutics
  30. How genetic modifiers influence the phenotype of spinal muscular atrophy and suggest future therapeutic approaches
    Brunhilde Wirth, Lutz Garbes, Markus Rießland · 2013 · Current Opinion in Genetics & Development
  31. Tandem hnRNP A1 RNA recognition motifs act in concert to repress the splicing of survival motor neuron exon 7
    Irene Beusch, Pierre Barraud, Ahmed Moursy, Antoine Cléry, Frédéric H.‐T. Allain · 2017 · eLife
  32. Gene delivery to the spinal cord using MRI-guided focused ultrasound
    Danielle Weber‐Adrian, Emmanuel Thévenot, M A O'Reilly, Wendy Oakden, Margarete K. Akens, Nicholas P. K. Ellens, Kelly Markham-Coultes, A Burgess, Joel A. Finkelstein, Albert Yee et al. · 2015 · Gene Therapy
  33. Translational Fidelity of Intrathecal Delivery of Self-Complementary AAV9–Survival Motor Neuron 1 for Spinal Muscular Atrophy
    Marco A. Passini, Jie Bu, Amy M. Richards, Christopher M. Treleaven, Jennifer A. Sullivan, Catherine R. O’Riordan, Abraham Scaria, Adrian P. Kells, Lluı́s Samaranch, Waldy San Sebastián et al. · 2014 · Human Gene Therapy
  34. Advances in spinal muscular atrophy therapeutics
    Valeria Parente, Stefania Paola Corti · 2018 · Therapeutic Advances in Neurological Disorders
  35. AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
    Rithu K. Pattali, Yongchao Mou, Xue-Jun Li · 2019 · Gene Therapy
  36. Local and retrograde gene transfer into primate neuronal pathways via adeno-associated virus serotype 8 and 9
    Yoshito Masamizu, Takashi Okada, Keisuke Kawasaki, Hidetoshi Ishibashi, Shinsuke Yuasa, Shin’ichi Takeda, Isao Hasegawa, Kiyoshi Nakahara · 2011 · Neuroscience
  37. Antisense Oligonucleotides for the Treatment of Spinal Muscular Atrophy
    Paul N. Porensky, Arthur H.M. Burghes · 2013 · Human Gene Therapy
  38. Two breakthrough gene-targeted treatments for spinal muscular atrophy: challenges remain
    Charlotte J. Sumner, Thomas O. Crawford · 2018 · Journal of Clinical Investigation
  39. Nucleic Acid Drugs—Current Status, Issues, and Expectations for Exosomes
    Yoji Yamada · 2021 · Cancers
  40. Feasibility and Safety of Systemic rAAV9-h NAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates
    Darren A. Murrey, Bartholomew J. Naughton, F. Jason Duncan, Aaron S. Meadows, Tierra A. Bobo, Katie Campbell, William G. Bremer, Christopher M. Walker, Laurie Goodchild, Brad N. Bolon et al. · 2014 · Human Gene Therapy Clinical Development
  41. Candidate Proteins, Metabolites and Transcripts in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study
    Richard S. Finkel, Thomas O. Crawford, Kathryn J. Swoboda, Petra Kaufmann, Péter Juhász, Xiaohong Li, Yu Feng Guo, Rebecca H. Li, Felicia L. Trachtenberg, Suzanne J. Forrest et al. · 2012 · PLoS ONE
  42. Spinal muscular atrophy: Development and implementation of potential treatments
    William David Arnold, Arthur H.M. Burghes · 2013 · Annals of Neurology
  43. Systemic restoration of UBA1 ameliorates disease in spinal muscular atrophy
    Rachael A. Powis, Evangelia Karyka, Penelope J Boyd, Julien Côme, Ross A. Jones, Yinan Zheng, Eva Szunyogová, Ewout J. N. Groen, Gillian Hunter, Derek Thomson et al. · 2016 · JCI Insight
  44. Time Is Motor Neuron: Therapeutic Window and Its Correlation with Pathogenetic Mechanisms in Spinal Muscular Atrophy
    Alessandra Govoni, Delia Gagliardi, Giacomo P. Comi, Stefania Paola Corti · 2018 · Molecular Neurobiology
  45. Prolactin increases SMN expression and survival in a mouse model of severe spinal muscular atrophy via the STAT5 pathway
    Faraz Farooq, Francisco Abadía‐Molina, Jeremiah Hadwen, Duncan MacKenzie, Luke Witherspoon, Matthew Osmond, Martin Holčı́k, Alex MacKenzie · 2011 · Journal of Clinical Investigation
  46. A lung tropic AAV vector improves survival in a mouse model of surfactant B deficiency
    Martin H. Kang, Laura P. van Lieshout, Liqun Xu, Jakob M. Domm, Arul Vadivel, Laurent Renesme, Christian Mühlfeld, Maria Hurskainen, Ivana Mižíková, Yanlong Pei et al. · 2020 · Nature Communications
  47. High-resolution melting: Applications in genetic disorders
    Tze‐Kiong Er, Jan‐Gowth Chang · 2012 · Clinica Chimica Acta
  48. Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9
    Deniz Dalkara, Leah C. Byrne, T Lee, Natalie Hoffmann, David V. Schaffer, John G. Flannery · 2011 · Gene Therapy
  49. Newborn screening for spinal muscular atrophy: Anticipating an imminent need
    Han C. Phan, Jennifer L. Taylor, Harry Hannon, R. Rodney Howell · 2015 · Seminars in Perinatology
  50. Sam68 binds Alu-rich introns in SMN and promotes pre-mRNA circularization
    Vittoria Pagliarini, Ariane Jolly, Pamela Bielli, Valentina Di Rosa, Pierre de la Grange, Claudio Sette · 2019 · Nucleic Acids Research
  51. Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model
    Lei Sheng, Frank Rigo, C. Frank Bennett, Adrian R. Krainer, Yimin Hua · 2020 · Nucleic Acids Research
  52. Mouse Survival Motor Neuron Alleles That Mimic SMN2 Splicing and Are Inducible Rescue Embryonic Lethality Early in Development but Not Late
    Suzan M. Hammond, Rocky G. Gogliotti, Vamshi K. Rao, Ariane Beauvais, Rashmi Kothary, Christine J. DiDonato · 2010 · PLoS ONE
  53. Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy
    Rachael A. Potter, Danielle A. Griffin, Patricia C. Sondergaard, R.W. Johnson, Eric R. Pozsgai, Kristin N. Heller, Ellyn L. Peterson, Kimmo K Lehtimäki, Hillarie Plessner Windish, Plavi Mittal et al. · 2017 · Human Gene Therapy
  54. RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy
    Chuhong Hu, Ronald W. Busuttil, Gerald S. Lipshutz · 2010 · The Journal of Gene Medicine
  55. Systemic gene delivery to the central nervous system using Adeno-associated virus
    Mathieu Bourdenx, Nathalie Dutheil, Erwan Bézard, Benjamin Dehay · 2014 · Frontiers in Molecular Neuroscience
  56. Altered Levels of MicroRNA-9, -206, and -132 in Spinal Muscular Atrophy and Their Response to Antisense Oligonucleotide Therapy
    Francesco Catapano, Irina T. Zaharieva, Mariacristina Scoto, Elena Marrosu, Jennifer Elizabeth Morgan, Francesco Muntoni A, Haiyan Zhou · 2016 · Molecular Therapy — Nucleic Acids
  57. Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice
    Jacob I. Ayers, Susan Fromholt, Olga Sinyavskaya, Zoe B. Siemienski, Awilda M. Rosario, Andrew Li, Keith W. Crosby, Pedro E. Cruz, Nadia DiNunno, Christopher Janus et al. · 2014 · Molecular Therapy
  58. Combination of SMN Trans -Splicing and a Neurotrophic Factor Increases the Life Span and Body Mass in a Severe Model of Spinal Muscular Atrophy
    Monir Shababi, Jacqueline Glascock, Christian L. Lorson · 2010 · Human Gene Therapy
  59. BBB-targeting, protein-based nanomedicines for drug and nucleic acid delivery to the CNS
    Hugo Peluffo, Ugutz Unzueta, María Luciana Negro‐Demontel, Zhikun Xu, Esther Váquez, Neus Ferrer‐Miralles, Antonio Villaverde · 2015 · Biotechnology Advances
  60. Intravenous Injections in Neonatal Mice
    Sara E. Gombash, Brian K. Kaspar, Kevin D. Foust · 2014 · Journal of Visualized Experiments
  61. Spinal Muscular Atrophy Patient iPSC-Derived Motor Neurons Have Reduced Expression of Proteins Important in Neuronal Development
    Heidi R. Fuller, Berhan Mandefro, Sally L. Shirran, Andrew R. Gross, Anjoscha S. Kaus, Catherine H. Botting, Glenn E. Morris, Dhruv Sareen · 2016 · Frontiers in Cellular Neuroscience
  62. Molecular Mechanisms of Neurodegeneration in Spinal Muscular Atrophy
    Saif Ahmad, Kanchan Bhatia, Annapoorna Kannan, L. Gangwani · 2016 · Journal of Experimental Neuroscience
  63. Myostatin inhibition in combination with antisense oligonucleotide therapy improves outcomes in spinal muscular atrophy
    Haiyan Zhou, Jinhong Meng, Alberto Malerba, Francesco Catapano, Palittiya Sintusek, Susan Jarmin, L. Feng, Ngoc Lu‐Nguyen, Lianwen Sun, Virginie Mariot et al. · 2020 · Journal of Cachexia Sarcopenia and Muscle
  64. Clinical Applications Involving CNS Gene Transfer
    Kantor, Boris, McCown, Thomas, Leone, Paola, Gray, Steven J. · 2014 · Advances in Genetics
  65. High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models
    Brahim Belbellaa, Laurence Reutenauer, Nadia Messaddeq, Laurent Monassier, Hélène M. Puccio · 2020 · Molecular Therapy — Methods & Clinical Development
  66. Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1
    Randy J. Chandler, Ian Martin Williams, Alana L. Gibson, Cristin D. Davidson, Arturo A. Incao, Brandon T. Hubbard, Forbes D. Porter, William J. Pavan, Charles P. Venditti · 2016 · Human Molecular Genetics
  67. Antisense Oligonucleotide-Based Therapies for Diseases Caused by pre-mRNA Processing Defects
    Frank Rigo, Punit P. Seth, C. Frank Bennett · 2014 · Advances in experimental medicine and biology
  68. Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors
    Devan L. Puhl, Anthony R. D’Amato, Ryan J. Gilbert · 2019 · Brain Research Bulletin
  69. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems
    Citra Nurfarah Zaini Mattar, Simon N. Waddington, Arijit Biswas, Nuryanti Binti Johana, Xuewei Ng, Angus S. Fisk, Nicholas M. Fisk, L G Tan, Ahad A. Rahim, Suzanne M. K. Buckley et al. · 2012 · Gene Therapy
  70. Improved Antisense Oligonucleotide Design to Suppress Aberrant SMN2 Gene Transcript Processing: Towards a Treatment for Spinal Muscular Atrophy
    Chalermchai Mitrpant, Paul N. Porensky, Haiyan Zhou, Loren L. Flynn, Francesco Muntoni A, Sue Fletcher, Steve Donald Wilton, Arthur H.M. Burghes · 2013 · PLoS ONE
  71. Partial restoration of cardio-vascular defects in a rescued severe model of spinal muscular atrophy
    Monir Shababi, Javad Habibi, Lixin Ma, Jacqueline Glascock, James R. Sowers, Christian L. Lorson · 2012 · Journal of Molecular and Cellular Cardiology
  72. Spinal muscular atrophy: An update on therapeutic progress
    Joonbae Seo, Matthew D. Howell, Natalia Nikolaevna Singh, Ravindra Narayan Singh · 2013 · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease
  73. The DcpS inhibitor RG3039 improves motor function in SMA mice
    James P. Van Meerbeke, Rebecca M. Gibbs, Heather L. Plasterer, Wenyan Miao, Zhihua Feng, Ming-Yi Lin, Agnieszka A. Rucki, Claribel D. Wee, Bing Xia, Shefali Sharma et al. · 2013 · Human Molecular Genetics
  74. Genome engineering: a new approach to gene therapy for neuromuscular disorders
    Christopher E. Nelson, Jacqueline N. Robinson-Hamm, Charles A. Gersbach · 2017 · Nature Reviews Neurology
  75. Therapy development for spinal muscular atrophy: perspectives for muscular dystrophies and neurodegenerative disorders
    Sibylle Jablonka, Luisa Hennlein, Michael Sendtner · 2022 · Neurological Research and Practice
  76. Giant axonal neuropathy: An updated perspective on its pathology and pathogenesis
    Bethany Johnson-Kerner, Lisa Anne Roth, J. Palmer Greene, Hynek Wichterle, Douglas M. Sproule · 2014 · Muscle & Nerve
  77. Electrophysiological biomarkers in spinal muscular atrophy: proof of concept
    William David Arnold, Paul N. Porensky, Vicki L. McGovern, Chitra C. Iyer, Sandra I. Duqué, Xiaobai Li, Kathrin Meyer, Leah Schmelzer, Brian K. Kaspar, Stephen J. Kolb et al. · 2013 · Annals of Clinical and Translational Neurology
  78. Genetic therapy for the nervous system
    William J. Bowers, Xandra O. Breakefield, Miguel Sena‐Esteves · 2011 · Human Molecular Genetics
  79. Is Spinal Muscular Atrophy a disease of the motor neurons only: pathogenesis and therapeutic implications?
    Chiara Simone, A. Ramirez, M. Bucchia, P. Rinchetti, H. Rideout, D. Papadimitriou, D. Re, S. Corti · 2015 · Cellular and Molecular Life Sciences
  80. The genetics of spinal muscular atrophies
    Claribel D. Wee, Lingling Kong, Charlotte J. Sumner · 2010 · Current Opinion in Neurology
  81. Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)
    Megan E. Bosch, Amy L. Aldrich, Rachel W. Fallet, Jessica Odvody, Maria E. Burkovetskaya, Kaitlyn Schuberth, Julie A. Fitzgerald, Kevin D. Foust, Tammy Kielian · 2016 · Journal of Neuroscience
  82. AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials
    Nathan Hardcastle, Nicholas M. Boulis, Thais Federici · 2017 · Expert Opinion on Biological Therapy
  83. Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases
    Della Grace Thomas Parambi, Khalid Saad Alharbi, Rajesh Kumar, Seetha Harilal, Gaber El‐Saber Batiha, Natália Alves Martins, Omnia Magdy Hendawy, Arafa Musa, Dibya Sundar Panda, Bijo Mathew · 2021 · Molecular Neurobiology
  84. Development of Therapeutic Splice-Switching Oligonucleotides
    Petra Disterer, Adrianna Kryczka, Yuqi Liu, Yusef Eamon Badi, Jessie J. Wong, James Stuart Owen, Bernard Chong Eu Khoo · 2014 · Human Gene Therapy
  85. Defects in pancreatic development and glucose metabolism in SMN-depleted mice independent of canonical spinal muscular atrophy neuromuscular pathology
    Mélissa Bowerman, John‐Paul Michalski, Ariane Beauvais, Lyndsay M. Murray, Yves DeRepentigny, Rashmi Kothary · 2014 · Human Molecular Genetics
  86. Spinal Muscular Atrophy: Past, Present, and Future
    Lainie Friedman Ross, Jennifer M. Kwon · 2019 · NeoReviews
  87. Expression of Muscle-Specific MiRNA 206 in the Progression of Disease in a Murine SMA Model
    Valeria Valsecchi, Marina Boido, Elena De Amicis, Antonio Piras, Alessandro Vercelli · 2015 · PLoS ONE
  88. Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy
    Jacqueline Glascock, Monir Shababi, Mary J. Wetz, Megan M. Krogman, Christian L. Lorson · 2011 · Biochemical and Biophysical Research Communications
  89. SMN expression is required in motor neurons to rescue electrophysiological deficits in the SMNΔ7 mouse model of SMA
    Vicki L. McGovern, Chitra C. Iyer, William David Arnold, Sara E. Gombash, Phillip G. Zaworski, Anton J. Blatnik, Kevin D. Foust, Arthur H.M. Burghes · 2015 · Human Molecular Genetics
  90. Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy
    Erkan Y. Osman, Pei–Fen Yen, Christian L. Lorson · 2011 · Molecular Therapy
  91. Evolving AAV-delivered therapeutics towards ultimate cures
    Xijing He, Brian Anugerah Urip, Zhenjie Zhang, Chun Christopher Ngan, Bo Feng · 2021 · Journal of Molecular Medicine
  92. Current Treatment Options in Neurology—SMA Therapeutics
    Megan A. Waldrop, Stephen J. Kolb · 2019 · Current Treatment Options in Neurology
  93. Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice
    Glascock, Jacqueline J., Osman, Erkan Y., Coady, Tristan H., Rose, Ferrill F., Shababi, Monir, Lorson, Christian L. · 2011 · Journal of Visualized Experiments
  94. Selective Vulnerability of Spinal and Cortical Motor Neuron Subpopulations in delta7 SMA Mice
    Paolo d’Errico, Marina Boido, Antonio Piras, Valeria Valsecchi, Elena De Amicis, Denise Locatelli, Silvia Capra, Francesco Vagni, Alessandro Vercelli, Giorgio Stefano Battaglia · 2013 · PLoS ONE
  95. New developments in exon skipping and splice modulation therapies for neuromuscular diseases
    Aleksander Touznik, Joshua J. A. Lee, Toshifumi Yokota · 2014 · Expert Opinion on Biological Therapy
  96. Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy
    Tejal Aslesh, Toshifumi Yokota · 2022 · Cells
  97. Genetic inhibition of JNK3 ameliorates spinal muscular atrophy
    Naresh K. Genabai, Saif Ahmad, Zhanying Zhang, Xiaoting Jiang, Cynthia A. Gabaldon, Laxman Gangwani · 2015 · Human Molecular Genetics
  98. Stasimon Contributes to the Loss of Sensory Synapses and Motor Neuron Death in a Mouse Model of Spinal Muscular Atrophy
    Christian M. Simon, Meaghan Van Alstyne, Francesco Lotti, Elena Bianchetti, Sarah Tisdale, Daniel Martin Watterson, George Z. Mentis, Livio Pellizzoni · 2019 · Cell Reports
  99. Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice
    Afrooz Rashnonejad, Gholamhossein Amini Chermahini, Cumhur Gündüz, Hüseyin Önay, Ayça Aykut, Burak Durmaz, Meral Baka, Qin Su, Guangping Gao, Ferda Özkınay · 2019 · Molecular Therapy
  100. Combinatorial Polycation Synthesis and Causal Machine Learning Reveal Divergent Polymer Design Rules for Effective pDNA and Ribonucleoprotein Delivery
    Ramya Kumar, Ngoc Le, Felipe Oviedo, Mary E. Brown, Theresa M. Reineke · 2022 · JACS Au