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Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni & Laurent Servais. (2022) Onasemnogene abeparvovec for the treatment of spinal muscular atrophy. Expert Opinion on Biological Therapy 22:9, pages 1075-1090.
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Hemanth R. Nelvagal & Jonathan D. Cooper. (2017) Translating preclinical models of neuronal ceroid lipofuscinosis: progress and prospects. Expert Opinion on Orphan Drugs 5:9, pages 727-740.
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Kristy Swiderski & Gordon S Lynch. (2015) Therapeutic potential of orphan drugs for the rare skeletal muscle diseases. Expert Opinion on Orphan Drugs 3:12, pages 1397-1425.
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Eric Hastie & R Jude Samulski. (2015) Recombinant adeno-associated virus vectors in the treatment of rare diseases. Expert Opinion on Orphan Drugs 3:6, pages 675-689.
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Longping V Tse, Sven Moller-Tank & Aravind Asokan. (2015) Strategies to circumvent humoral immunity to adeno-associated viral vectors. Expert Opinion on Biological Therapy 15:6, pages 845-855.
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Articles from other publishers (94)
Thomas Lee Moore, Giovanna Pannuzzo, Gabriella Costabile, Anna Lisa Palange, Raffaele Spanò, Miguel Ferreira, Adriana Carol Eleonora Graziano, Paolo Decuzzi & Venera Cardile. (2023) Nanomedicines to treat rare neurological disorders: The case of Krabbe disease. Advanced Drug Delivery Reviews 203, pages 115132.
Crossref
Crossref
Francisco Ramírez, Jiaqi Wu, Charles Haitjema & Chris Heger. (2023) Development of a highly sensitive imaged cIEF immunoassay for studying AAV capsid protein charge heterogeneity. ELECTROPHORESIS 44:15-16, pages 1258-1266.
Crossref
Crossref
Venla Soini, Gudrun Schreiber, Bernd Wilken & Anna Kathrin Hell. (2023) Early Development of Spinal Deformities in Children Severely Affected with Spinal Muscular Atrophy after Gene Therapy with Onasemnogene Abeparvovec—Preliminary Results. Children 10:6, pages 998.
Crossref
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Troy T Rohn, Dean Radin, Tracy Brandmeyer, Barry J Linder, Emile Andriambeloson, Stéphanie Wagner, James Kehler, Ana Vasileva, Huaien Wang, John L Mee & James H Fallon. (2023)
Genetic modulation of the
HTR2A
gene reduces anxiety-related behavior in mice
. PNAS Nexus 2:6.
Crossref
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Amber M. Tetlow, Brianna M. Jackman, Mohammed M. Alhadidy, Patricia Muskus, David G. Morgan & Marcia N. Gordon. (2023) Neural atrophy produced by AAV tau injections into hippocampus and anterior cortex of middle-aged mice. Neurobiology of Aging 124, pages 39-50.
Crossref
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Yueyi LiHongyu ZengYuhao WeiXuelei MaZhiyao He. (2023) An Overview of the Therapeutic Strategies for the Treatment of Spinal Muscular Atrophy. Human Gene Therapy 34:5-6, pages 180-191.
Crossref
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Sen Yan, Xiao Zheng, Yingqi Lin, Caijuan Li, Zhaoming Liu, Jiawei Li, Zhuchi Tu, Yu Zhao, Chunhui Huang, Yizhi Chen, Jun Li, Xichen Song, Bofeng Han, Wei Wang, Weien Liang, Liangxue Lai, Xiao-Jiang Li & Shihua Li. (2023) Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington’s disease. Nature Biomedical Engineering 7:5, pages 629-646.
Crossref
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Alisa A. Shaimardanova, Valeriya V. Solovyeva, Shaza S. Issa & Albert A. Rizvanov. (2023) Gene Therapy of Sphingolipid Metabolic Disorders. International Journal of Molecular Sciences 24:4, pages 3627.
Crossref
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Anett Hudák, Matthew Roach, Dávid Pusztai, Aladár Pettkó-Szandtner, Annamária Letoha, László Szilák, Mimoun Azzouz & Tamás Letoha. (2023) Syndecan-4 Mediates the Cellular Entry of Adeno-Associated Virus 9. International Journal of Molecular Sciences 24:4, pages 3141.
Crossref
Crossref
Jeong-A Lim, Priya S. Kishnani & Baodong Sun. (2022) Suppression of pullulanase-induced cytotoxic T cell response with a dual promoter in GSD IIIa mice. JCI Insight 7:23.
Crossref
Crossref
Huijun Yuan, Keith A. Webster, Muhammad Tariq Bhatti, William W. Hauswirth, Alfred S. Lewin & John Guy. (2022) Amelioration of Leigh syndrome induced by mouse blastocyst complementation with a mutant human mitochondrial ATP synthase 6. Clinical and Translational Discovery 2:3.
Crossref
Crossref
Jiaqi Wu & Chris Heger. (2022) Establishment of a Platform Imaged Capillary Isoelectric Focusing (icIEF) Characterization Method for Adeno-Associated Virus (AAV) Capsid Proteins. Green Analytical Chemistry, pages 100027.
Crossref
Crossref
Yunseon Yang, Min-Jong Seok, Ye Eun Kim, Yunjung Choi, Jae-Jin Song, Yanuar Alan Sulistio, Seong-hoon Kim, Mi-Yoon Chang, Soo-Jin Oh, Min-Ho Nam, Yun Kyung Kim, Tae-Gyun Kim, Heh-In Im, Seong-Ho Koh & Sang-Hun Lee. (2022) Adeno-associated virus (AAV) 9-mediated gene delivery of Nurr1 and Foxa2 ameliorates symptoms and pathologies of Alzheimer disease model mice by suppressing neuro-inflammation and glial pathology. Molecular Psychiatry.
Crossref
Crossref
Jaspreet Kaur & Rune W. Berg. (2022) Viral strategies for targeting spinal neuronal subtypes in adult wild-type rodents. Scientific Reports 12:1.
Crossref
Crossref
Lisa M. Riedmayr, Klara S. Hinrichsmeyer, Nina Karguth, Sybille Böhm, Victoria Splith, Stylianos Michalakis & Elvir Becirovic. (2022) dCas9-VPR-mediated transcriptional activation of functionally equivalent genes for gene therapy. Nature Protocols 17:3, pages 781-818.
Crossref
Crossref
Xintao Zhang, Zheng Chai, Amanda Lee Dobbins, Michelle S. Itano, Charles Askew, Zhe Miao, Hongqian Niu, R. Jude Samulski & Chengwen Li. (2022) Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain. Biomaterials 281, pages 121340.
Crossref
Crossref
Della Grace Thomas Parambi, Khalid Saad Alharbi, Rajesh Kumar, Seetha Harilal, Gaber El-Saber Batiha, Natália Cruz-Martins, Omnia Magdy, Arafa Musa, Dibya Sundar Panda & Bijo Mathew. (2021) Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases. Molecular Neurobiology 59:1, pages 191-233.
Crossref
Crossref
Ceri A. Pickering & Nicholas D. Mazarakis. (2021) Viral Vector Delivery of DREADDs for CNS Therapy. Current Gene Therapy 21:3, pages 191-206.
Crossref
Crossref
Hongliang Gong, Nini Yuan, Zhiming Shen, Cheng Tang, Stewart Shipp, Liling Qian, Yiliang Lu, Ian Max Andolina, Shenghai Zhang, Jihong Wu, Hui Yang & Wei Wang. (2021) Transduction catalysis: Doxorubicin amplifies rAAV-mediated gene expression in the cortex of higher-order vertebrates. iScience 24:6, pages 102685.
Crossref
Crossref
Manisha Singh, Surinder P. Singh, Deepshikha Yadav, Mugdha Agarwal, Shriya Agarwal, Vinayak Agarwal, Geeta Swargiary, Sahil Srivastava, Sakshi Tyagi, Ramneek Kaur & Shalini Mani. (2021) Targeted Delivery for Neurodegenerative Disorders Using Gene Therapy Vectors: Gene Next Therapeutic Goals. Current Gene Therapy 21:1, pages 23-42.
Crossref
Crossref
Antonio Luchicchi, Tommy Pattij, John Noel M. Viaña, Sybren de Kloet & Nathan Marchant. (2021) Tracing goes viral: Viruses that introduce expression of fluorescent proteins in chemically-specific neurons. Journal of Neuroscience Methods 348, pages 109004.
Crossref
Crossref
Jeong-A Lim, Su Jin Choi, Fengqin Gao, Priya S. Kishnani & Baodong Sun. (2020) A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme. Molecular Therapy - Methods & Clinical Development 18, pages 240-249.
Crossref
Crossref
Yeon-Suk Yang, Jun Xie, Sachin Chaugule, Dan Wang, Jung-Min Kim, JiHea Kim, Phillip W.L. Tai, Seok-kyo Seo, Ellen Gravallese, Guangping Gao & Jae-Hyuck Shim. (2020) Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy. Molecular Therapy - Methods & Clinical Development 17, pages 922-935.
Crossref
Crossref
Venu Talla, Rajeshwari Koilkonda & John Guy. (2020) Gene Therapy with Single-Subunit Yeast NADH-Ubiquinone Oxidoreductase (NDI1) Improves the Visual Function in Experimental Autoimmune Encephalomyelitis (EAE) Mice Model of Multiple Sclerosis (MS). Molecular Neurobiology 57:4, pages 1952-1965.
Crossref
Crossref
Gwladys Gernoux, Alisha M. Gruntman, Meghan Blackwood, Marina Zieger, Terence R. Flotte & Christian Mueller. (2020) Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans. Molecular Therapy 28:3, pages 747-757.
Crossref
Crossref
David C. Schorling, Astrid Pechmann & Janbernd Kirschner. (2020) Advances in Treatment of Spinal Muscular Atrophy – New Phenotypes, New Challenges, New Implications for Care. Journal of Neuromuscular Diseases 7:1, pages 1-13.
Crossref
Crossref
Shinji Kubota, Wupuer Sidikejiang, Moeko Kudo, Ken‐ichi Inoue, Tatsuya Umeda, Masahiko Takada & Kazuhiko Seki. (2019) Optogenetic recruitment of spinal reflex pathways from large‐diameter primary afferents in non‐transgenic rats transduced with AAV9/Channelrhodopsin 2. The Journal of Physiology 597:19, pages 5025-5040.
Crossref
Crossref
Yeon-Suk Yang, Jun Xie, Dan Wang, Jung-Min Kim, Phillip W. L. Tai, Ellen Gravallese, Guangping Gao & Jae-Hyuck Shim. (2019) Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. Nature Communications 10:1.
Crossref
Crossref
Kurt Whittemore, Aksinya Derevyanko, Paula Martinez, Rosa Serrano, Martí Pumarola, Fàtima Bosch & Maria A. Blasco. (2019) Telomerase gene therapy ameliorates the effects of neurodegeneration associated to short telomeres in mice. Aging 11:10, pages 2916-2948.
Crossref
Crossref
Rohan S. Balkawade, Chao Chen, Michael R. Crowley, David K. Crossman, William L. Clapp, Jill W. Verlander & Caroline B. Marshall. (2019) Podocyte-specific expression of Cre recombinase promotes glomerular basement membrane thickening. American Journal of Physiology-Renal Physiology 316:5, pages F1026-F1040.
Crossref
Crossref
Jenny Lange, Luke J. Haslett, Emyr Lloyd-Evans, Jennifer M. Pocock, Mark S. Sands, Brenda P. Williams & Jonathan D. Cooper. (2018) Compromised astrocyte function and survival negatively impact neurons in infantile neuronal ceroid lipofuscinosis. Acta Neuropathologica Communications 6:1.
Crossref
Crossref
Lindsey M. Snyder, Michael C. Chiang, Emanuel Loeza-Alcocer, Yu Omori, Junichi Hachisuka, Tayler D. Sheahan, Jenna R. Gale, Peter C. Adelman, Elizabeth I. Sypek, Stephanie A. Fulton, Robert L. Friedman, Margaret C. Wright, Melissa Giraldo Duque, Yeon Sun Lee, Zeyu Hu, Huizhen Huang, Xiaoyun Cai, Kimberly A. Meerschaert, Vidhya Nagarajan, Toshiro Hirai, Gregory Scherrer, Daniel H. Kaplan, Frank Porreca, Brian M. Davis, Michael S. Gold, H. Richard Koerber & Sarah E. Ross. (2018) Kappa Opioid Receptor Distribution and Function in Primary Afferents. Neuron 99:6, pages 1274-1288.e6.
Crossref
Crossref
Robert D. Dayton, Mychal S. Grames & Ronald L. Klein. (2018) More expansive gene transfer to the rat CNS: AAV PHP.EB vector dose–response and comparison to AAV PHP.B. Gene Therapy 25:5, pages 392-400.
Crossref
Crossref
Jeff L. EllsworthMichael O'CallaghanHillard RubinAlbert Seymour. (2018) Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans. Human Gene Therapy Clinical Development 29:1, pages 60-67.
Crossref
Crossref
Jeff L EllsworthMichael OCallaghanHillard RubinAlbert Seymour. (2018) Low Seroprevalence of Neutralizing Antibodies Targeting Two Clade F AAV in Humans. Human Gene Therapy Clinical Development.
Crossref
Crossref
HuiQian Lin, HaoJie Hu, WeiSong Duan, YaLing Liu, GuoJun Tan, ZhongYao Li, YaKun Liu, BinBin Deng, XueQin Song, Wan Wang, Di Wen, Ying Wang & ChunYan Li. (2016) Intramuscular Delivery of scAAV9-hIGF1 Prolongs Survival in the hSOD1G93A ALS Mouse Model via Upregulation of D-Amino Acid Oxidase. Molecular Neurobiology 55:1, pages 682-695.
Crossref
Crossref
Qi Wu, Yong Han & Qingchun Tong. 2018. Neural Regulation of Metabolism. Neural Regulation of Metabolism
211
233
.
Sun H. Park, Matthew R. Eber, Shunsuke Tsuzuki, Mary E. Booker, Aaron G. Sunil, D. Brooke Widner, Renee A. Parker, Christopher M. Peters & Yusuke Shiozawa. (2017) Adeno-associated virus serotype rh10 is a useful gene transfer vector for sensory nerves that innervate bone in immunodeficient mice. Scientific Reports 7:1.
Crossref
Crossref
Lilei Yu, Liping Zhou, Gang Cao, Sunny S. Po, Bing Huang, Xiaoya Zhou, Menglong Wang, Shenxu Yuan, Zhuo Wang, Songyun Wang & Hong Jiang. (2017) Optogenetic Modulation of Cardiac Sympathetic Nerve Activity to Prevent Ventricular Arrhythmias. Journal of the American College of Cardiology 70:22, pages 2778-2790.
Crossref
Crossref
Jayanth S. Chandran, Paul S. Sharp, Evangelia Karyka, João Miguel da Conceição Aves-Cruzeiro, Ian Coldicott, Lydia Castelli, Guillaume Hautbergue, Mark O. Collins & Mimoun Azzouz. (2017) Site Specific Modification of Adeno-Associated Virus Enables Both Fluorescent Imaging of Viral Particles and Characterization of the Capsid Interactome. Scientific Reports 7:1.
Crossref
Crossref
Charles ShyngHemanth R. NelvagalJoshua T. DearbornJaana TyyneläRobert E. SchmidtMark S. SandsJonathan D. Cooper. (2017) Synergistic effects of treating the spinal cord and brain in CLN1 disease. Proceedings of the National Academy of Sciences 114:29.
Crossref
Crossref
J J Siu, N J Queen, W Huang, F Q Yin, X Liu, C Wang, D M McTigue & L Cao. (2017) Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes. Gene Therapy 24:6, pages 361-369.
Crossref
Crossref
Jessica M. Kelly, Allison Bradbury, Douglas R. Martin & Mark E. Byrne. (2017) Emerging therapies for neuropathic lysosomal storage disorders. Progress in Neurobiology 152, pages 166-180.
Crossref
Crossref
GA Dissen, K Adachi, A Lomniczi, T Chatkupt, BL Davidson, H Nakai & SR Ojeda. (2017) Engineering a gene silencing viral construct that targets the cat hypothalamus to induce permanent sterility: An update. Reproduction in Domestic Animals 52, pages 354-358.
Crossref
Crossref
Corey L. Williams, Cedric R. Uytingco, Warren W. Green, Jeremy C. McIntyre, Kirill Ukhanov, Arthur D. Zimmerman, Dana T. Shively, Lian Zhang, Darryl Y. Nishimura, Val C. Sheffield & Jeffrey R. Martens. (2017) Gene Therapeutic Reversal of Peripheral Olfactory Impairment in Bardet-Biedl Syndrome. Molecular Therapy 25:4, pages 904-916.
Crossref
Crossref
K. Castillo, V. Valenzuela, M. Oñate & C. Hetz. 2017. Molecular Characterization of Autophagic Responses, Part B. Molecular Characterization of Autophagic Responses, Part B
109
131
.
S.S. Sunshine, J. Jarecki, A. MacKenzie & K.S. Chen. 2017. Spinal Muscular Atrophy. Spinal Muscular Atrophy
263
281
.
Cary O. Harding. 2017. Inherited Metabolic Diseases. Inherited Metabolic Diseases
155
171
.
Alessandra Ricca & Angela Gritti. (2016) Perspective on innovative therapies for globoid cell leukodystrophy. Journal of Neuroscience Research 94:11, pages 1304-1317.
Crossref
Crossref
Yangyang Huang & Cheryl F. Dreyfus. (2016) The role of growth factors as a therapeutic approach to demyelinating disease. Experimental Neurology 283, pages 531-540.
Crossref
Crossref
P Chapdelaine, C Gérard, N Sanchez, K Cherif, J Rousseau, D L Ouellet, D Jauvin & J P Tremblay. (2016) Development of an AAV9 coding for a 3XFLAG-TALEfrat#8-VP64 able to increase in vivo the human frataxin in YG8R mice. Gene Therapy 23:7, pages 606-614.
Crossref
Crossref
Jakob Körbelin, Godwin Dogbevia, Stefan Michelfelder, Dirk A Ridder, Agnes Hunger, Jan Wenzel, Henning Seismann, Melanie Lampe, Jacqueline Bannach, Manolis Pasparakis, Jürgen A Kleinschmidt, Markus Schwaninger & Martin Trepel. (2016) A brain microvasculature endothelial cell‐specific viral vector with the potential to treat neurovascular and neurological diseases. EMBO Molecular Medicine 8:6, pages 609-625.
Crossref
Crossref
A Donsante, Z McEachin, J Riley, C H Leung, L Kanz, D M O'Connor & N M Boulis. (2016) Intracerebroventricular delivery of self-complementary adeno-associated virus serotype 9 to the adult rat brain. Gene Therapy 23:5, pages 401-407.
Crossref
Crossref
Megan S. Keiser, Holly B. Kordasiewicz & Jodi L. McBride. (2016) Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia. Human Molecular Genetics 25:R1, pages R53-R64.
Crossref
Crossref
Julian C. Assmann, Jakob Körbelin & Markus Schwaninger. (2016) Genetic manipulation of brain endothelial cells in vivo. Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease 1862:3, pages 381-394.
Crossref
Crossref
Georg von Jonquieres, Dominik Fröhlich, Claudia B. Klugmann, Xin Wen, Anne E. Harasta, Roshini Ramkumar, Ziggy H. T. Spencer, Gary D. Housley & Matthias Klugmann. (2016) Recombinant Human Myelin-Associated Glycoprotein Promoter Drives Selective AAV-Mediated Transgene Expression in Oligodendrocytes. Frontiers in Molecular Neuroscience 9.
Crossref
Crossref
Vera Lukashchuk, Katherine E Lewis, Ian Coldicott, Andrew J Grierson & Mimoun Azzouz. (2016) AAV9-mediated central nervous system–targeted gene delivery via cisterna magna route in mice. Molecular Therapy - Methods & Clinical Development 3, pages 15055.
Crossref
Crossref
Sara E. Gombash & Kevin D. Foust. 2016. Gene Therapy for Neurological Disorders. Gene Therapy for Neurological Disorders
231
237
.
Giridhar Murlidharan, R. Jude Samulski & Aravind Asokan. 2016. Translational Neuroscience. Translational Neuroscience
9
32
.
Carlotta Lambertini, Domenico Ventrella, Francesca Barone, Nicolina Cristina Sorrentino, Francesco Dondi, Alessandro Fraldi, Massimo Giunti, Enrico Maria Surace, Maria Laura Bacci & Noemi Romagnoli. (2015) Transdermal spinal catheter placement in piglets: Description and validation of the technique. Journal of Neuroscience Methods 255, pages 17-21.
Crossref
Crossref
Dominic Schomberg, Gurwattan Miranpuri, Tyler Duellman, Andrew Crowell, Raghu Vemuganti & Daniel Resnick. (2015) Spinal cord injury induced neuropathic pain: Molecular targets and therapeutic approaches. Metabolic Brain Disease 30:3, pages 645-658.
Crossref
Crossref
Kasey L. Jackson, Robert D. Dayton, Jeanne M. Fisher-Perkins, Peter J. Didier, Kate C. Baker, Maria Weimer, Amparo Gutierrez, Cooper D. Cain, J. Michael Mathis, Michael A. Gitcho, Bruce A. Bunnell & Ronald L. Klein. (2015) Initial gene vector dosing for studying symptomatology of amyotrophic lateral sclerosis in non-human primates. Journal of Medical Primatology 44:2, pages 66-75.
Crossref
Crossref
Han C. Phan, Jennifer L. Taylor, Harry Hannon & Rodney Howell. (2015) Newborn screening for spinal muscular atrophy: Anticipating an imminent need. Seminars in Perinatology 39:3, pages 217-229.
Crossref
Crossref
Yu Song & Weidong Pan. (2014) Exploration of the Pathogenesis of Amyotrophic Lateral Sclerosis from the Perspective of Motor Neuron TDP-43 Protein Expression and ADAR2 Activity. Integrative Medicine International 1:2, pages 119-125.
Crossref
Crossref
Jacob I Ayers, Susan Fromholt, Olga Sinyavskaya, Zoe Siemienski, Awilda M Rosario, Andrew Li, Keith W Crosby, Pedro E Cruz, Nadia M DiNunno, Christopher Janus, Carolina Ceballos-Diaz, David R Borchelt, Todd E Golde, Paramita Chakrabarty & Yona Levites. (2015) Widespread and Efficient Transduction of Spinal Cord and Brain Following Neonatal AAV Injection and Potential Disease Modifying Effect in ALS Mice. Molecular Therapy 23:1, pages 53-62.
Crossref
Crossref
K L Jackson, R D Dayton, E A Orchard, S Ju, D Ringe, G A Petsko, L E Maquat & R L Klein. (2014) Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis. Gene Therapy 22:1, pages 20-28.
Crossref
Crossref
Tsuneya Ikezu. 2015. Cognitive Enhancement. Cognitive Enhancement
111
137
.
Maya Woodbury, Tomomi Kiyota & Tsuneya Ikezu. 2015. Gene Delivery and Therapy for Neurological Disorders. Gene Delivery and Therapy for Neurological Disorders
85
120
.
R. Jude SamulskiNicholas Muzyczka. (2014) AAV-Mediated Gene Therapy for Research and Therapeutic Purposes. Annual Review of Virology 1:1, pages 427-451.
Crossref
Crossref
Giridhar Murlidharan, Richard J. Samulski & Aravind Asokan. (2014) Biology of adeno-associated viral vectors in the central nervous system. Frontiers in Molecular Neuroscience 7.
Crossref
Crossref
Pádraig J. MulcahyKayleigh IremongerEvangelia KarykaSaúl Herranz-MartínKa-To ShumJanice Kal Van TamMimoun Azzouz. (2014) Gene Therapy: A Promising Approach to Treating Spinal Muscular Atrophy. Human Gene Therapy 25:7, pages 575-586.
Crossref
Crossref
Jesse R. McLean, Gaynor A. Smith, Emily M. Rocha, Melissa A. Hayes, Jonathan A. Beagan, Penelope J. Hallett & Ole Isacson. (2014) Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection. Neuroscience Letters 576, pages 73-78.
Crossref
Crossref
Daniel J. Schuster, Jaclyn A. Dykstra, Maureen S. Riedl, Kelley F. Kitto, Lalitha R. Belur, R. Scott McIvor, Robert P. Elde, Carolyn A. Fairbanks & Lucy Vulchanova. (2014) Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse. Frontiers in Neuroanatomy 8.
Crossref
Crossref
Mathieu Bourdenx, Nathalie Dutheil, Erwan Bezard & Benjamin Dehay. (2014) Systemic gene delivery to the central nervous system using Adeno-associated virus. Frontiers in Molecular Neuroscience 7.
Crossref
Crossref
Matthew D Howell, Natalia N Singh & Ravindra N Singh. (2014) Advances in therapeutic development for spinal muscular atrophy. Future Medicinal Chemistry 6:9, pages 1081-1099.
Crossref
Crossref
Juliana Navarro-Yepes, Laura Zavala-Flores, Annadurai Anandhan, Fang Wang, Maciej Skotak, Namas Chandra, Ming Li, Aglaia Pappa, Daniel Martinez-Fong, Luz Maria Del Razo, Betzabet Quintanilla-Vega & Rodrigo Franco. (2014) Antioxidant gene therapy against neuronal cell death. Pharmacology & Therapeutics 142:2, pages 206-230.
Crossref
Crossref
W Lerchner, B Corgiat, V Der Minassian, R C Saunders & B J Richmond. (2014) Injection parameters and virus dependent choice of promoters to improve neuron targeting in the nonhuman primate brain. Gene Therapy 21:3, pages 233-241.
Crossref
Crossref
Maya E. Woodbury & Tsuneya Ikezu. (2013) Fibroblast Growth Factor-2 Signaling in Neurogenesis and Neurodegeneration. Journal of Neuroimmune Pharmacology 9:2, pages 92-101.
Crossref
Crossref
G P Swain, M Prociuk, J H Bagel, P O'Donnell, K Berger, K Drobatz, B L Gurda, M E Haskins, M S Sands & C H Vite. (2013) Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain. Gene Therapy 21:1, pages 28-36.
Crossref
Crossref
Lee Ying, Xavier Matabosch, Montserrat Serra, Berna Watson, Cedric Shackleton & Gordon Watson. (2014) Biochemical and physiological improvement in a mouse model of Smith–Lemli–Opitz syndrome (SLOS) following gene transfer with AAV vectors. Molecular Genetics and Metabolism Reports 1, pages 103-113.
Crossref
Crossref
Mathias Mahn, Shiri Ron & Ofer Yizhar. 2014. Viral Vector Approaches in Neurobiology and Brain Diseases. Viral Vector Approaches in Neurobiology and Brain Diseases
289
310
.
Yaohui Tang, Yaning Li, Xiaojie Lin, Peng Miao, Yongting Wang & Guo-Yuan Yang. 2014. Cerebral Angiogenesis. Cerebral Angiogenesis
317
329
.
Takenari Yamashita, Hui Lin Chai, Sayaka Teramoto, Shoji Tsuji, Kuniko Shimazaki, Shin‐ichi Muramatsu & Shin Kwak. (2013)
Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous
AAV
9‐
ADAR
2
delivery to motor neurons
. EMBO Molecular Medicine 5:11, pages 1710-1719.
Crossref
Crossref
Abdelwahed Chtarto, Olivier Bockstael, Terence Tshibangu, Olivier Dewitte, Marc Levivier & Liliane Tenenbaum. (2013) A next step in adeno-associated virus-mediated gene therapy for neurological diseases: regulation and targeting. British Journal of Clinical Pharmacology 76:2, pages 217-232.
Crossref
Crossref
Casey A Maguire, Matheus HW Crommentuijn, Dakai Mu, Eloise Hudry, Alberto Serrano-Pozo, Bradley T Hyman & Bakhos A Tannous. (2013) Mouse Gender Influences Brain Transduction by Intravascularly Administered AAV9. Molecular Therapy 21:8, pages 1470-1471.
Crossref
Crossref
Ghanashyam D. Ghadge, John D. Pavlovic, Sujatha P. Koduvayur, Brian K. Kay & Raymond P. Roos. (2013) Single chain variable fragment antibodies block aggregation and toxicity induced by familial ALS-linked mutant forms of SOD1. Neurobiology of Disease 56, pages 74-78.
Crossref
Crossref
Robert D Dayton, Michael A Gitcho, Elysse A Orchard, Jon D Wilson, David B Wang, Cooper D Cain, Jeffrey A Johnson, Yong-Jie Zhang, Leonard Petrucelli, J Michael Mathis & Ronald L Klein. (2013) Selective Forelimb Impairment in Rats Expressing a Pathological TDP-43 25 kDa C-terminal Fragment to Mimic Amyotrophic Lateral Sclerosis. Molecular Therapy 21:7, pages 1324-1334.
Crossref
Crossref
Erika Harno, Elizabeth C. Cottrell & Anne White. (2013) Metabolic Pitfalls of CNS Cre-Based Technology. Cell Metabolism 18:1, pages 21-28.
Crossref
Crossref
Georg von Jonquieres, Nadine Mersmann, Claudia Bettina Klugmann, Anne Editha Harasta, Beat Lutz, Orla Teahan, Gary David Housley, Dominik Fröhlich, Eva-Maria Krämer-Albers & Matthias Klugmann. (2013) Glial Promoter Selectivity following AAV-Delivery to the Immature Brain. PLoS ONE 8:6, pages e65646.
Crossref
Crossref
Hironori Okada, Hidetoshi Ishibashi, Hiromi Hayashita-Kinoh, Tomoko Chiyo, Yuko Nitahara-Kasahara, Yukihiro Baba, Sumiko Watanabe, Shin'ichi Takeda & Takashi Okada. (2013) Robust Long-term Transduction of Common Marmoset Neuromuscular Tissue With rAAV1 and rAAV9. Molecular Therapy - Nucleic Acids 2, pages e95.
Crossref
Crossref
Kewal K. JainKewal K. Jain. 2013. Applications of Biotechnology in Neurology. Applications of Biotechnology in Neurology
383
476
.
. (2012) Current World Literature. Current Opinion in Pediatrics 24:6, pages 770-779.
Crossref
Crossref
Emmanuel ThévenotJessica F. JordãoMeaghan A. O'ReillyKelly MarkhamYing-Qi WengKevin D. FoustBrian K. KasparKullervo HynynenIsabelle Aubert. (2012) Targeted Delivery of Self-Complementary Adeno-Associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-Guided Focused Ultrasound. Human Gene Therapy 23:11, pages 1144-1155.
Crossref
Crossref
Barry J. ByrneDarin J. FalkNathalie ClémentCathryn S. Mah. (2012) Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment. Human Gene Therapy 23:8, pages 808-815.
Crossref
Crossref