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Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS
Title Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS Author Bravo-Hernandez Mariana Co-authors Tadokoro T. Navarro M. Platoshyn Oleksandr Kobayashi Yoshiomi Marsala Silvia Miyanohara A Juhas Stefan Juhasova Jana Skalnikova Helena Tomori Zoltán 1956- SAVEXFYZ - Ústav experimentálnej fyziky SAV RID Vanický Ivo 1962 SAVNEBIO ; SAVBIOMED - Neurobiologický ústav SAV ORCID Studenovska Hana Proks Vladimír Chen PeiXi Govea-Perez Noe Ditsworth Dara Ciacci Joseph D. Gao Shang Zhu Wenlian Ahrens Eric T Driscoll Shawn P Glenn Thomas D McAlonis-Downes Melissa Da Cruz Sandrine Pfaff Samuel L. Kasper Brian K. Cleveland Don W. Marsala Martin SAVNEBIO ; SAVBIOMED - Neurobiologický ústav SAV Source document Nature medicine. Vol. 26, no. 1 (2020), p.118-130 Language eng - English Country US - United States of America Document kind rozpis článkov z periodík (rbx) Citations DUDMAN, Jessica - QI, Xin. Stress Granule Dysregulation in Amyotrophic Lateral Sclerosis. In FRONTIERS IN CELLULAR NEUROSCIENCE, 2020, vol. 14, no., pp. GUISSART, Claire - MOUZAT, Kevin - KANTAR, Jovana - LOUVEAU, Baptiste - VILQUIN, Paul - POLGE, Anne - RAOUL, Cedric - LUMBROSO, Serge. Premature termination codons in SOD1 causing Amyotrophic Lateral Sclerosis are predicted to escape the nonsense-mediated mRNA decay. In SCIENTIFIC REPORTS. ISSN 2045-2322, 2020, vol. 10, no. 1, pp. FRANKLIN, John P. - AZZOUZ, Mimoun - SHAW, Pamela J. SOD1-targeting therapies for neurodegenerative diseases: a review of current findings and future potential. In EXPERT OPINION ON ORPHAN DRUGS. ISSN 2167-8707, 2020, vol. 8, no. 10, pp. 379-392. KIM, Garam - GAUTIER, Olivia - TASSONI-TSUCHIDA, Eduardo - MA, X. Rosa - GITLER, Aaron D. ALS Genetics: Gains, Losses, and Implications for Future Therapies. In NEURON. ISSN 0896-6273, 2020, vol. 108, no. 5, pp. 822-842. NUNEZ-PERALTA, Claudia - ALONSO-PEREZ, Jorge - DIAZ-MANERA, Jordi. The increasing role of muscle MRI to monitor changes over time in untreated and treated muscle diseases. In CURRENT OPINION IN NEUROLOGY. ISSN 1350-7540, 2020, vol. 33, no. 5, pp. 611-620. SHEN, Yi - CAMPBELL, Robert E. - COTE, Daniel C. - PAQUET, Marie-Eve. Challenges for Therapeutic Applications of Opsin-Based Optogenetic Tools in Humans. In FRONTIERS IN NEURAL CIRCUITS, 2020, vol. 14, no., pp. MALIK, Ravinder - WIEDAU, Martina. Therapeutic Approaches Targeting Protein Aggregation in Amyotrophic Lateral Sclerosis. In FRONTIERS IN MOLECULAR NEUROSCIENCE. ISSN 1662-5099, 2020, vol. 13, no., pp. QIU, Junxiong - PENG, Peng - XIN, Min - WEN, Zhenkang - CHEN, Zhong - LIN, Sipeng - KUANG, Manyuan - FU, Yuan - FANG, Guibin - LI, Shixun - LI, Changchuan - MAO, Jiaji - QIN, Ling - DING, Yue. ZBTB20-mediated titanium particle-induced peri-implant osteolysis by promoting macrophage inflammatory responses. In BIOMATERIALS SCIENCE. ISSN 2047-4830, 2020, vol. 8, no. 11, pp. 3147-3163. MEROLA, Aristide - VAN LAAR, Amber - LONSER, Russell - BANKIEWICZ, Krzysztof. Gene therapy for Parkinson's disease: contemporary practice and emerging concepts. In EXPERT REVIEW OF NEUROTHERAPEUTICS. ISSN 1473-7175, 2020, vol. 20, no. 6, pp. 577-590. BORDONI, Matteo - SCARIAN, Eveljn - REY, Federica - GAGLIARDI, Stella - CARELLI, Stephana - PANSARASA, Orietta - CEREDA, Cristina. Biomaterials in Neurodegenerative Disorders: A Promising Therapeutic Approach. In INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2020, vol. 21, no. 9, pp. YERBURY, Justin J. - FARRAWELL, Natalie E. - MCALARY, Luke. Proteome Homeostasis Dysfunction: A Unifying Principle in ALS Pathogenesis. In TRENDS IN NEUROSCIENCES. ISSN 0166-2236, 2020, vol. 43, no. 5, pp. 274-284. ABATI, Elena - BRESOLIN, Nereo - COMI, Giacomo - CORTI, Stefania. Silence superoxide dismutase 1 (SOD1): a promising therapeutic target for amyotrophic lateral sclerosis (ALS). In EXPERT OPINION ON THERAPEUTIC TARGETS. ISSN 1472-8222, 2020, vol. 24, no. 4, pp. 295-310. GENC, Baris - GOZUTOK, Oge - KOCAK, Nuran - OZDINLER, P. Hande. The Timing and Extent of Motor Neuron Vulnerability in ALS Correlates with Accumulation of Misfolded SOD1 Protein in the Cortex and in the Spinal Cord. In CELLS, 2020, vol. 9, no. 2, pp. MORELLI, Kathryn H. - HATTON, Courtney L. - HARPER, Scott Q. - BURGESS, Robert W. Gene therapies for axonal neuropathies: Available strategies, successes to date, and what to target next. In BRAIN RESEARCH. ISSN 0006-8993, 2020, vol. 1732, no., pp. OZDINLER, P. Hande - GAUTAM, Mukesh - GOZUTOK, Oge - KONRAD, Csaba - MANFREDI, Giovanni - AREA GOMEZ, Estela - MITSUMOTO, Hiroshi - ERB, Marcella L. - TIAN, Zheng - HAASE, Georg. Better understanding the neurobiology of primary lateral sclerosis. In AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION. ISSN 2167-8421, 2020, vol. 21, no., pp. 35-46. MCALARY, L. - YERBURY, J. J. - CASHMAN, N. R. The prion-like nature of amyotrophic lateral sclerosis. In PRIONS AND NEURODEGENERATIVE DISEASES. ISSN 1877-1173, 2020, vol. 175, no., pp. 261-296. LUKACOVA, Nadezda - KISUCKA, Alexandra - KISS BIMBOVA, Katarina - BACOVA, Maria - ILENINOVA, Maria - KURUC, Tomas - GALIK, Jan. Glial-Neuronal Interactions in Pathogenesis and Treatment of Spinal Cord Injury. In INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, vol. 22, no. 24, pp. Dostupné na: https://doi.org/10.3390/ijms222413577. KAGIAVA, A. - RICHTER, J. - TRYFONOS, C. - LEAL-JULIA, M. - SARGIANNIDOU, I. - CHRISTODOULOU, C. - BOSCH, A. - KLEOPA, K. A. Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery. In SCIENTIFIC REPORTS. ISSN 2045-2322, 2021, vol. 11, no. 1, pp. Dostupné na: https://doi.org/10.1038/s41598-021-02694-1. BONIFACINO, Tiziana - ZERBO, Roberta Arianna - BALBI, Matilde - TORAZZA, Carola - FRUMENTO, Giulia - FEDELE, Ernesto - BONANNO, Giambattista - MILANESE, Marco. Nearly 30 Years of Animal Models to Study Amyotrophic Lateral Sclerosis: A Historical Overview and Future Perspectives. In INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, vol. 22, no. 22, pp. Dostupné na: https://doi.org/10.3390/ijms222212236. JENSEN, Thomas Leth - GOTZSCHE, Casper Rene - WOLDBYE, David P. D. Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord. In FRONTIERS IN MOLECULAR NEUROSCIENCE. ISSN 1662-5099, 2021, vol. 14, no., pp. Dostupné na: https://doi.org/10.3389/fnmol.2021.695937. BLIZARD, Sarah - PARK, Danielle - O'TOOLE, Natalie - NOROOZ, Sheeva - DELA TORRE, Martin - SON, Young - HOLSTEIN, Adam - AUSTIN, Scarlett - HARMAN, Joshua - HARASZTI, Samantha - FARED, Daved - XU, Mei. Neuron-Specific IMP2 Overexpression by Synapsin Promoter-Driven AAV9: A Tool to Study Its Role in Axon Regeneration. In CELLS, 2021, vol. 10, no. 10, pp. Dostupné na: https://doi.org/10.3390/cells10102654. TORRILLAS DE LA CAL, Alejandro - PANIAGUA-TORIJA, Beatriz - AREVALO-MARTIN, Angel - GUY FAULKES, Christopher - JESUS JIMENEZ, Antonio - FERRER, Isidre - MOLINA-HOLGADO, Eduardo - GARCIA-OVEJERO, Daniel. The Structure of the Spinal Cord Ependymal Region in Adult Humans Is a Distinctive Trait among Mammals. In CELLS, 2021, vol. 10, no. 9, pp. Dostupné na: https://doi.org/10.3390/cells10092235. XU, Xiaojiao - SHEN, Dingding - GAO, Yining - ZHOU, Qinming - NI, You - MENG, Huanyu - SHI, Hongqin - LE, Weidong - CHEN, Shengdi - CHEN, Sheng. A perspective on therapies for amyotrophic lateral sclerosis: can disease progression be curbed? In TRANSLATIONAL NEURODEGENERATION. ISSN 2047-9158, 2021, vol. 10, no. 1, pp. Dostupné na: https://doi.org/10.1186/s40035-021-00250-5. FAJARDO-SERRANO, Ana - RICO, Alberto J. - RODA, Elvira - HONRUBIA, Adriana - ARRIETA, Sandra - ARIZNABARRETA, Goiaz - CHOCARRO, Julia - LORENZO-RAMOS, Elena - PEJENAUTE, Alvaro - VAZQUEZ, Alfonso - LUIS LANCIEGO, Jose. Adeno-Associated Viral Vectors as Versatile Tools for Parkinson's Research, Both for Disease Modeling Purposes and for Therapeutic Uses. In INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2021, vol. 22, no. 12, pp. Dostupné na: https://doi.org/10.3390/ijms22126389. MAYL, Keith - SHAW, Christopher E. - LEE, Youn-Bok. Disease Mechanisms and Therapeutic Approaches in C9orf72 ALS-FTD. In BIOMEDICINES, 2021, vol. 9, no. 6, pp. Dostupné na: https://doi.org/10.3390/biomedicines9060601. KHORKOVA, Olga - HSIAO, Jane - WAHLESTEDT, Claes. Nucleic Acid-Based Therapeutics in Orphan Neurological Disorders: Recent Developments. In FRONTIERS IN MOLECULAR BIOSCIENCES, 2021, vol. 8, no., pp. Dostupné na: https://doi.org/10.3389/fmolb.2021.643681. AMADO, Defne A. - DAVIDSON, Beverly L. Gene therapy for ALS: A review. In MOLECULAR THERAPY. ISSN 1525-0016, 2021, vol. 29, no. 12, pp. 3345-3358. Dostupné na: https://doi.org/10.1016/j.ymthe.2021.04.008. ACIOGLU, Cigdem - LI, Lun - ELKABES, Stella. Contribution of astrocytes to neuropathology of neurodegenerative diseases. In BRAIN RESEARCH. ISSN 0006-8993, 2021, vol. 1758, no., pp. Dostupné na: https://doi.org/10.1016/j.brainres.2021.147291. KAGIAVA, Alexia - KARAISKOS, Christos - RICHTER, Jan - TRYFONOS, Christina - JENNINGS, Matthew J. - HESLEGRAVE, Amanda J. - SARGIANNIDOU, Irene - STAVROU, Marina - ZETTERBERG, Henrik - REILLY, Mary M. - CHRISTODOULOU, Christina - HORVATH, Rita - KLEOPA, Kleopas A. AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy. In GENE THERAPY. ISSN 0969-7128, 2021, vol. 28, no. 10-11, pp. 659-675. Dostupné na: https://doi.org/10.1038/s41434-021-00250-0. HUNANYAN, Arsen S. - KANTOR, Boris - PURANAM, Ram S. - ELLIOTT, Courtney - MCCALL, Angela - DHINDSA, Justin - PAGADALA, Promila - WALLACE, Keri - POE, Jordan - GUNDUZ, Talha - ASOKAN, Aravind - KOEBERL, Dwight D. - ELMALLAH, Mai K. - MIKATI, Mohamad A. Adeno-Associated Virus-Mediated Gene Therapy in the Mashlool, Atp1a3(Mashl/+), Mouse Model of Alternating Hemiplegia of Childhood. In HUMAN GENE THERAPY. ISSN 1043-0342, 2021, vol. 32, no. 7-8, pp. 405-419. Dostupné na: https://doi.org/10.1089/hum.2020.191. NISTAL-VILLAN, Estanislao - ARGEMI, Josepmaria - DE JAIME-SOGUERO, Anchel - FERRERO, Roberto - DI SCALA, Marianna - RODRIGUEZ-GARCIA, Estefania - COLL, Aniol - RIUS-ROCABERT, Sergio - PRIETO, Jesus - GONZALEZ-ASEGUINOLAZA, Gloria - ARAGON, Tomas. Linking the Expression of Therapeutic Genes to Unfolded Protein Response: A New Option for Anti-Hepatitis B Virus Gene Therapy. In HUMAN GENE THERAPY. ISSN 1043-0342, 2021, vol. 32, no. 7-8, pp. 341-348. Dostupné na: https://doi.org/10.1089/hum.2019.336. PIGUET, Francoise - DENIS, Timothee de Saint - AUDOUARD, Emilie - BECCARIA, Kevin - ANDRE, Arthur - WURTZ, Guillaume - SCHATZ, Raphael - ALVES, Sandro - SEVIN, Caroline - ZERAH, Michel - CARTIER, Nathalie. The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System. In HUMAN GENE THERAPY. ISSN 1043-0342, 2021, vol. 32, no. 7-8, pp. 349-374. Dostupné na: https://doi.org/10.1089/hum.2020.105. CAPPELLA, Marisa - PRADAT, Pierre-Francois - QUERIN, Giorgia - BIFERI, Maria Grazia. Beyond the Traditional Clinical Trials for Amyotrophic Lateral Sclerosis and The Future Impact of Gene Therapy. In JOURNAL OF NEUROMUSCULAR DISEASES. ISSN 2214-3599, 2021, vol. 8, no. 1, pp. 25-38. Dostupné na: https://doi.org/10.3233/JND-200531. AMADO, Defne A. - DAVIDSON, Beverly L. Gene therapy for ALS: A review. In Molecular Therapy. ISSN 15250016, 2021-12-01, 29, 12, pp. 3345-3358. Dostupné na: https://doi.org/10.1016/j.ymthe.2021.04.008. WANG, S.S. - ICHINOMIYA, T. - SAVCHENKO, P. - WANG, D.S. - SAWADA, A. - LI, X.J. - DUONG, T. - LI, W.X. - BONDS, J.A. - KIM, E.J. - MIYANOHARA, A. - ROTH, D.M. - PATEL, H.H. - PATEL, P.M. - TADOKORO, T. - MARSALA, M. - HEAD, B.P. Subpial delivery of adeno-associated virus 9-synapsin- caveolin-1 (iAAV9/i-iSynCavl/i) preserves motor neuron and neuromuscular junction morphology, motor function, delays disease onset, and extends survival in hSOD1SUPG93A/SUP mice. In THERANOSTICS. ISSN 1838-7640, 2022, vol. 12, no. 12, p. 5389-5403. Dostupné na: https://doi.org/10.7150/thno.72614. BENATTI, H.R. - GRAY-EDWARDS, H.L. Adeno-Associated Virus Delivery Limitations for Neurological Indications. In HUMAN GENE THERAPY. ISSN 1043-0342, JAN 1 2022, vol. 33, no. 1-2, p. 1-7. Dostupné na: https://doi.org/10.1089/hum.2022.29196.hrb. ODEH, H.M. - FARE, C.M. - SHORTER, J. Nuclear-Import Receptors Counter Deleterious Phase Transitions in Neurodegenerative Disease. In JOURNAL OF MOLECULAR BIOLOGY. ISSN 0022-2836, JAN 15 2022, vol. 434, no. 1. Dostupné na: https://doi.org/10.1016/j.jmb.2021.167220. OYA, R. - TSUKAMOTO, O. - HITSUMOTO, T. - NAKAHARA, N. - OKAMOTO, C. - MATSUOKA, K. - KATO, H. - INOHARA, H. - TAKASHIMA, S. Gene Transfer of Skeletal Muscle-Type Myosin Light Chain Kinase via Adeno-Associated Virus 6 Improves Muscle Functions in an Amyotrophic Lateral Sclerosis Mouse Model. In INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. FEB 2022, vol. 23, no. 3. Dostupné na: https://doi.org/10.3390/ijms23031747. MIKI, S. - KOGA, T. - MCKINNEY, A.M. - PARISIAN, A.D. - TADOKORO, T. - VADLA, R. - MASALA, M. - HEVNER, R.F. - COSTELLO, J.F. - FURNARI, F. iTERT/i promoter C228T mutation in neural progenitors confers growth advantage following telomere shortening iin vivo/i. In NEURO-ONCOLOGY. ISSN 1522-8517, DEC 1 2022, vol. 24, no. 12, p. 2063-2075. Dostupné na: https://doi.org/10.1093/neuonc/noac080. GOSSET, P. - CAMU, W. - RAOUL, C. - MEZGHRANI, A. Prionoids in amyotrophic lateral sclerosis. In BRAIN COMMUNICATIONS. MAY 2 2022, vol. 4, no. 3. Dostupné na: https://doi.org/10.1093/braincomms/fcac145. KHANG, M. - BINDRA, R.S. - SALTZMAN, W.M. Intrathecal delivery and its applications in leptomeningeal disease. In ADVANCED DRUG DELIVERY REVIEWS. ISSN 0169-409X, JUL 2022, vol. 186. Dostupné na: https://doi.org/10.1016/j.addr.2022.114338. MARLIN, E. - VIU-IDOCIN, C. - ARRASATE, M. - ARAGóN, T. The Role and Therapeutic Potential of the Integrated Stress Response in Amyotrophic Lateral Sclerosis. In INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES. JUL 2022, vol. 23, no. 14. Dostupné na: https://doi.org/10.3390/ijms23147823. SURDYKA, M. - JESION, E. - NIEWIADOMSKA-CIMICKA, A. - TROTTIER, Y. - KALINOWSKA-POSKA, Z. - FIGIEL, M. Selective transduction of cerebellar Purkinje and granule neurons using delivery of AAV-PHP.eB and AAVrh10 vectors at axonal terminal locations. In FRONTIERS IN MOLECULAR NEUROSCIENCE. ISSN 1662-5099, SEP 13 2022, vol. 15. Dostupné na: https://doi.org/10.3389/fnmol.2022.947490. LIU, P.P. - DAI, S.K. - MI, T.W. - TANG, G.B. - WANG, Z. - WANG, H. - DU, H.Z. - TANG, Y. - TENG, Z.Q. - LIU, C.M. Acetate supplementation restores cognitive deficits caused by ARID1A haploinsufficiency in excitatory neurons. In EMBO MOLECULAR MEDICINE. ISSN 1757-4676, DEC 7 2022, vol. 14, no. 12. Dostupné na: https://doi.org/10.15252/emmm.202215795. JAMBEAU, M. - MEYER, K.D. - HRUSKA-PLOCHAN, M. - TABET, R. - LEE, C.Z. - RAY-SONI, A. - AGUILAR, C. - SAVAGE, K. - MISHRA, N. - CAVEGN, N. - BORTER, P. - LIN, C.C. - JANSEN-WEST, K.R. - JIANG, J. - FREYERMUTH, F. - LIA, N. - DE ROSSI, P. - PéREZ-BERLANGA, M. - JIANG, X. - DAUGHRITY, L.M. - PEREIRA, J. - NARAYANAN, S. - GU, Y.Z. - DHOKAI, S. - DALKILIC-LIDDLE, I. - MANIECKA, Z. - WEBER, J. - WORKMAN, M. - MCALONIS-DOWNES, M. - BEREZOVSKI, E. - ZHANG, Y.J. - BERRY, J. - WAINGER, B.J. - KANKEL, M.W. - RUSHE, M. - HOCK, C. - NITSCH, R.M. - CLEVELAND, D.W. - PETRUCELLI, L. - GENDRON, T.F. - MONTRASIO, F. - GRIMM, J. - POLYMENIDOU, M. - LAGIER-TOURENNE, C. Comprehensive evaluation of human-derived anti-poly-GA antibodies in cellular and animal models of iC9orf72/i disease. In PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. ISSN 0027-8424, DEC 6 2022, vol. 119, no. 49. Dostupné na: https://doi.org/10.1073/pnas.2123487119. GERMAIN, N.D. - CHUNG, W.K. - SARMIERE, P.D. RNA interference (RNAi)-based therapeutics for treatment of rare neurologic diseases. In MOLECULAR ASPECTS OF MEDICINE. ISSN 0098-2997, JUN 2023, vol. 91. Dostupné na: https://doi.org/10.1016/j.mam.2022.101148. KONDO, T. - INOUE, I. - UMEYAMA, K. - WATANABE, M. - MATSUNARI, H. - UCHIKURA, A. - NAKANO, K. - TSUKITA, K. - IMAMURA, K. - NAGASHIMA, H. - INOUE, H. A Transgenic Pig Model With Human Mutanti SOD1/i Exhibits the Early Pathology of Amyotrophic Lateral Sclerosis. In LABORATORY INVESTIGATION. ISSN 0023-6837, FEB 2023, vol. 103, no. 2. Dostupné na: https://doi.org/10.1016/j.labinv.2022.100013. CAMU, W. - DE LA CRUZ, E. - ESSELIN, F. Therapeutic tools for familial ALS. In REVUE NEUROLOGIQUE. ISSN 0035-3787, JAN-FEB 2023, vol. 179, no. 1-2, p. 49-53. Dostupné na: https://doi.org/10.1016/j.neurol.2022.10.001. MERJANE, J. - CHUNG, R.G. - PATANI, R. - LISOWSKI, L. Molecular mechanisms of amyotrophic lateral sclerosis as broad therapeutic targets for gene therapy applications utilizing adeno-associated viral vectors. In MEDICINAL RESEARCH REVIEWS. ISSN 0198-6325, JUL 2023, vol. 43, no. 4, p. 829-854. Dostupné na: https://doi.org/10.1002/med.21937. GAO, T.C. - HUO, J. - XIN, C. - YANG, J. - LIU, Q. - DONG, H. - LI, R. - LIU, Y.L. Protective effects of intrathecal injection of AAV9-RabGGTB-GFPSUP+/SUP in SOD1SUPG93A/SUP mice. In FRONTIERS IN AGING NEUROSCIENCE. ISSN 1663-4365, MAR 14 2023, vol. 15. Dostupné na: https://doi.org/10.3389/fnagi.2023.1092607. STAVROU, M. - KAGIAVA, A. - SARGIANNIDOU, I. - GEORGIOU, E. - KLEOPA, K.A. Charcot-Marie-Tooth neuropathies: Current gene therapy advances and the route toward translation. In JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM. ISSN 1085-9489, JUN 2023, vol. 28, no. 2, p. 150-168. Dostupné na: https://doi.org/10.1111/jns.12543. DUAN, W.Z. - URANI, E. - MATTSON, M.P. The potential of gene editing for Huntington's disease. In TRENDS IN NEUROSCIENCES. ISSN 0166-2236, MAY 2023, vol. 46, no. 5, p. 365-376. Dostupné na: https://doi.org/10.1016/j.tins.2023.02.005. XIE, S.S. - GUAN, C.B. - HUANG, T.M. - YANG, G. - HU, J.Z. - SUN, D.Y. - LU, H.B. Activating Mitochondrial Sirtuin 3 in Chondrocytes Alleviates Aging-Induced Fibrocartilage Layer Degeneration and Promotes Healing of Degenerative Rotator Cuff Injury. In AMERICAN JOURNAL OF PATHOLOGY. ISSN 0002-9440, JUL 2023, vol. 193, no. 7, p. 939-949. Dostupné na: https://doi.org/10.1016/j.ajpath.2023.03.013. BAKAVAYEV, S. - STAVSKY, A. - ARGUETI-OSTROVSKY, S. - YEHEZKEL, G. - FRIDMANN-SIRKIS, Y. - BARAK, Z. - GITLER, D. - ISRAELSON, A. - ENGEL, S. Blocking an epitope of misfolded SOD1 ameliorates disease phenotype in a model of amyotrophic lateral sclerosis. In BRAIN. ISSN 0006-8950, 2023 JUL 3 2023. Dostupné na: https://doi.org/10.1093/brain/awad222. TOSOLINI, A.P. - SMITH, G.M. Editorial: Gene therapy for the central and peripheral nervous system, volume II. In FRONTIERS IN MOLECULAR NEUROSCIENCE. ISSN 1662-5099, AUG 1 2023, vol. 16. Dostupné na: https://doi.org/10.3389/fnmol.2023.1258458. FANG, Y.M. - CHEN, W.C. - ZHENG, W.J. - YANG, Y.S. - ZHANG, Y. - CHEN, X.L. - PEI, M.Q. - LIN, S. - HE, H.F. A cutting-edge strategy for spinal cord injury treatment: resident cellular transdifferentiation. In FRONTIERS IN CELLULAR NEUROSCIENCE. AUG 30 2023, vol. 17. Dostupné na: https://doi.org/10.3389/fncel.2023.1237641. ZHU, L.H. - LI, S.H. - LI, X.J. - YIN, P. Pathological insights from amyotrophic lateral sclerosis animal models: comparisons, limitations, and challenges. In TRANSLATIONAL NEURODEGENERATION. ISSN 2047-9158, SEP 20 2023, vol. 12, no. 1. Dostupné na: https://doi.org/10.1186/s40035-023-00377-7. MADUKA, C.V. - ALHAJ, M. - URAL, E. - HABEEB, O.M. - KUHNERT, M.M. - SMITH, K. - MAKELA, A.V. - POPE, H. - CHEN, S. - HIX, J.M. - MALLETT, C.L. - CHUNG, S.J. - HAKUN, M. - TUNDO, A. - ZINN, K.R. - HANKENSON, K.D. - GOODMAN, S.B. - NARAYAN, R. - CONTAG, C.H. Polylactide Degradation Activates Immune Cells by Metabolic Reprogramming. In ADVANCED SCIENCE. 2023 SEP 22 2023. Dostupné na: https://doi.org/10.1002/advs.202304632. GUAN, T. - ZHOU, T. - ZHANG, X.S. - GUO, Y. - YANG, C.X. - LIN, J.S. - ZHANG, J.V. - CHENG, Y.Q. - MARZBAN, H. - WANG, Y.T. - KONG, J.M. Selective removal of misfolded SOD1 delays disease onset in a mouse model of amyotrophic lateral sclerosis. In CELLULAR AND MOLECULAR LIFE SCIENCES. ISSN 1420-682X, OCT 2023, vol. 80, no. 10. Dostupné na: https://doi.org/10.1007/s00018-023-04956-9. MARLIN, E. - VALENCIA, M. - PEREGRíN, N. - FERRERO, R. - NICOLáS, M.J. - VINUEZA-GAVILANES, R. - PINEDA-LUCENA, A. - ARTIEDA, J. - ARRASATE, M. - ARAGóN, T. Pharmacological inhibition of the integrated stress response accelerates disease progression in an amyotrophic lateral sclerosis mouse model. In BRITISH JOURNAL OF PHARMACOLOGY. ISSN 0007-1188, 2024 FEB 2024, vol. 181, no. 3, p. 495-508. Dostupné na: https://doi.org/10.1111/bph.16260. BRADFORD, W.H. - ZHANG, J. - GUTIERREZ-LARA, E.J. - LIANG, Y. - DO, A. - WANG, T.M. - NGUYEN, L. - MATARAARACHCHI, N. - WANG, J. - GU, Y.S. - MCCULLOCH, A. - PETERSON, K.L. - SHEIKH, F. Plakophilin 2 gene therapy prevents and rescues arrhythmogenic right ventricular cardiomyopathy in a mouse model harboring patient genetics. In NATURE CARDIOVASCULAR RESEARCH. DEC 2023, vol. 2, no. 12, p. 1246-+. Dostupné na: https://doi.org/10.1038/s44161-023-00370-3. WEN, D. - JI, Y.X. - LI, Y.Y. - DUAN, W.S. - WANG, Y.Y. - LI, Z.Y. - TAO, M.C. - LIU, Y.K. iOPTN/i gene therapy increases autophagy and protects mitochondria in iSOD1/i-G93A-expressing transgenic mice and cells. In FEBS JOURNAL. ISSN 1742-464X, 2023 DEC 1 2023. Dostupné na: https://doi.org/10.1111/febs.17009. GEORGIOU, E. - KAGIAVA, A. - SARGIANNIDOU, I. - SCHIZA, N. - STAVROU, M. - RICHTER, J. - TRYFONOS, C. - ZETTERBERG, H. - HESLEGRAVE, A. - CHRISTODOULOU, C. - KLEOPA, K.A. AAV9-mediated SH3TC2 gene replacement therapy targeted to Schwann cells for the treatment of CMT4C. In MOLECULAR THERAPY. ISSN 1525-0016, 2023 NOV 1 2023, vol. 31, no. 11, p. 3290-3307. Dostupné na: https://doi.org/10.1016/j.ymthe.2023.08.020. Category ADCA - Scientific papers in foreign journals registered in Current Contents Connect with IF (impacted) Category of document (from 2022) V3 - Vedecký výstup publikačnej činnosti z časopisu Type of document článok Year 2020 Registered in WOS Registered in SCOPUS Registered in CCC DOI 10.1038/s41591-019-0674-1 
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File name Access Size Downloaded Type License Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS .pdf available 21.1 MB 12 Publisher's version rok CC IF IF Q (best) JCR Av Jour IF Perc SJR SJR Q (best) CiteScore A rok vydania rok metriky IF IF Q (best) SJR SJR Q (best) 2020 2019 36.130 Q1 15.812 Q1
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