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Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP. Mendell JR, et al. Mol Ther. 2021 Feb 3;29(2):464-488. doi: 10.1016/j.ymthe.2020.12.007. Epub 2020 Dec 10. Mol Ther. 2021. PMID: 33309881 Free PMC article. Review.
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular myopathy [XLMTM]; and diseases of the central nervous system, including Alzheimer's disease, Parkinson's disease, Canavan disease, aromatic …
We review gene therapy for neuromuscular disorders (spinal muscular atrophy [SMA]; Duchenne muscular dystrophy [DMD]; X-linked myotubular my …
Standards of Care for the Health of Transgender and Gender Diverse People, Version 8.
Coleman E, Radix AE, Bouman WP, Brown GR, de Vries ALC, Deutsch MB, Ettner R, Fraser L, Goodman M, Green J, Hancock AB, Johnson TW, Karasic DH, Knudson GA, Leibowitz SF, Meyer-Bahlburg HFL, Monstrey SJ, Motmans J, Nahata L, Nieder TO, Reisner SL, Richards C, Schechter LS, Tangpricha V, Tishelman AC, Van Trotsenburg MAA, Winter S, Ducheny K, Adams NJ, Adrián TM, Allen LR, Azul D, Bagga H, Başar K, Bathory DS, Belinky JJ, Berg DR, Berli JU, Bluebond-Langner RO, Bouman MB, Bowers ML, Brassard PJ, Byrne J, Capitán L, Cargill CJ, Carswell JM, Chang SC, Chelvakumar G, Corneil T, Dalke KB, De Cuypere G, de Vries E, Den Heijer M, Devor AH, Dhejne C, D'Marco A, Edmiston EK, Edwards-Leeper L, Ehrbar R, Ehrensaft D, Eisfeld J, Elaut E, Erickson-Schroth L, Feldman JL, Fisher AD, Garcia MM, Gijs L, Green SE, Hall BP, Hardy TLD, Irwig MS, Jacobs LA, Janssen AC, Johnson K, Klink DT, Kreukels BPC, Kuper LE, Kvach EJ, Malouf MA, Massey R, Mazur T, McLachlan C, Morrison SD, Mosser SW, Neira PM, Nygren U, Oates JM, Obedin-Maliver J, Pagkalos G, Patton J, Phanuphak N, Rachlin K, Reed T, Rider GN, Ristori J, Robbins-Cherry S, Roberts SA, Rodriguez-Wallberg KA, Rosenthal SM, Sabir K, Safer JD, Scheim A… See abstract for full author list ➔ Coleman E, et al. Int J Transgend Health. 2022 Sep 6;23(Suppl 1):S1-S259. doi: 10.1080/26895269.2022.2100644. eCollection 2022. Int J Transgend Health. 2022. PMID: 36238954 Free PMC article.
Connecting copper and cancer: from transition metal signalling to metalloplasia.
Ge EJ, Bush AI, Casini A, Cobine PA, Cross JR, DeNicola GM, Dou QP, Franz KJ, Gohil VM, Gupta S, Kaler SG, Lutsenko S, Mittal V, Petris MJ, Polishchuk R, Ralle M, Schilsky ML, Tonks NK, Vahdat LT, Van Aelst L, Xi D, Yuan P, Brady DC, Chang CJ. Ge EJ, et al. Nat Rev Cancer. 2022 Feb;22(2):102-113. doi: 10.1038/s41568-021-00417-2. Epub 2021 Nov 11. Nat Rev Cancer. 2022. PMID: 34764459 Free PMC article. Review.
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Speed C, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Griggs RC; FOR-DMD Investigators of the Muscle Study Group; Straub V, van Ruiten H, Childs AM, Ciafaloni E, Shieh PB, Spinty S, Maggi L, Baranello G, Butterfield RJ, Horrocks IA, Roper H, Alhaswani Z, Flanigan KM, Kuntz NL, Manzur A, Darras BT, Kang PB, Morrison L, Krzesniak-Swinarska M, Mah JK, Mongini TE, Ricci F, von der Hagen M, Finkel RS, O'Reardon K, Wicklund M, Kumar A, McDonald CM, Han JJ, Joyce N, Henricson EK, Schara-Schmidt U, Gangfuss A, Wilichowski E, Barohn RJ, Statland JM, Campbell C, Vita G, Vita GL, Howard JF Jr, Hughes I, McMillan HJ, Pegoraro E, Bello L, Burnette WB, Thangarajh M, Chang T. Guglieri M, et al. JAMA. 2022 Apr 19;327(15):1456-1468. doi: 10.1001/jama.2022.4315. JAMA. 2022. PMID: 35381069 Free PMC article. Clinical Trial.
Z-form extracellular DNA is a structural component of the bacterial biofilm matrix.
Buzzo JR, Devaraj A, Gloag ES, Jurcisek JA, Robledo-Avila F, Kesler T, Wilbanks K, Mashburn-Warren L, Balu S, Wickham J, Novotny LA, Stoodley P, Bakaletz LO, Goodman SD. Buzzo JR, et al. Cell. 2021 Nov 11;184(23):5740-5758.e17. doi: 10.1016/j.cell.2021.10.010. Epub 2021 Nov 3. Cell. 2021. PMID: 34735796 Free PMC article.
Impaired Human Cardiac Cell Development due to NOTCH1 Deficiency.
Ye S, Wang C, Xu Z, Lin H, Wan X, Yu Y, Adhicary S, Zhang JZ, Zhou Y, Liu C, Alonzo M, Bi J, Ramirez-Navarro A, Deschenes I, Ma Q, Garg V, Wu JC, Zhao MT. Ye S, et al. Circ Res. 2023 Jan 20;132(2):187-204. doi: 10.1161/CIRCRESAHA.122.321398. Epub 2022 Dec 30. Circ Res. 2023. PMID: 36583388 Free PMC article.
Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV.
Naeimi Kararoudi M, Likhite S, Elmas E, Yamamoto K, Schwartz M, Sorathia K, de Souza Fernandes Pereira M, Sezgin Y, Devine RD, Lyberger JM, Behbehani GK, Chakravarti N, Moriarity BS, Meyer K, Lee DA. Naeimi Kararoudi M, et al. Cell Rep Methods. 2022 Jun 13;2(6):100236. doi: 10.1016/j.crmeth.2022.100236. eCollection 2022 Jun 20. Cell Rep Methods. 2022. PMID: 35784645 Free PMC article.
Apoptotic cell death in disease-Current understanding of the NCCD 2023.
Vitale I, Pietrocola F, Guilbaud E, Aaronson SA, Abrams JM, Adam D, Agostini M, Agostinis P, Alnemri ES, Altucci L, Amelio I, Andrews DW, Aqeilan RI, Arama E, Baehrecke EH, Balachandran S, Bano D, Barlev NA, Bartek J, Bazan NG, Becker C, Bernassola F, Bertrand MJM, Bianchi ME, Blagosklonny MV, Blander JM, Blandino G, Blomgren K, Borner C, Bortner CD, Bove P, Boya P, Brenner C, Broz P, Brunner T, Damgaard RB, Calin GA, Campanella M, Candi E, Carbone M, Carmona-Gutierrez D, Cecconi F, Chan FK, Chen GQ, Chen Q, Chen YH, Cheng EH, Chipuk JE, Cidlowski JA, Ciechanover A, Ciliberto G, Conrad M, Cubillos-Ruiz JR, Czabotar PE, D'Angiolella V, Daugaard M, Dawson TM, Dawson VL, De Maria R, De Strooper B, Debatin KM, Deberardinis RJ, Degterev A, Del Sal G, Deshmukh M, Di Virgilio F, Diederich M, Dixon SJ, Dynlacht BD, El-Deiry WS, Elrod JW, Engeland K, Fimia GM, Galassi C, Ganini C, Garcia-Saez AJ, Garg AD, Garrido C, Gavathiotis E, Gerlic M, Ghosh S, Green DR, Greene LA, Gronemeyer H, Häcker G, Hajnóczky G, Hardwick JM, Haupt Y, He S, Heery DM, Hengartner MO, Hetz C, Hildeman DA, Ichijo H, Inoue S, Jäättelä M, Janic A, Joseph B, Jost PJ, Kanneganti TD, Karin M, Kashkar H, Kaufmann T, Kelly … See abstract for full author list ➔ Vitale I, et al. Cell Death Differ. 2023 May;30(5):1097-1154. doi: 10.1038/s41418-023-01153-w. Epub 2023 Apr 26. Cell Death Differ. 2023. PMID: 37100955 Free PMC article. Review.
25th Annual Computational Neuroscience Meeting: CNS-2016.
Sharpee TO, Destexhe A, Kawato M, Sekulić V, Skinner FK, Wójcik DK, Chintaluri C, Cserpán D, Somogyvári Z, Kim JK, Kilpatrick ZP, Bennett MR, Josić K, Elices I, Arroyo D, Levi R, Rodriguez FB, Varona P, Hwang E, Kim B, Han HB, Kim T, McKenna JT, Brown RE, McCarley RW, Choi JH, Rankin J, Popp PO, Rinzel J, Tabas A, Rupp A, Balaguer-Ballester E, Maturana MI, Grayden DB, Cloherty SL, Kameneva T, Ibbotson MR, Meffin H, Koren V, Lochmann T, Dragoi V, Obermayer K, Psarrou M, Schilstra M, Davey N, Torben-Nielsen B, Steuber V, Ju H, Yu J, Hines ML, Chen L, Yu Y, Kim J, Leahy W, Shlizerman E, Birgiolas J, Gerkin RC, Crook SM, Viriyopase A, Memmesheimer RM, Gielen S, Dabaghian Y, DeVito J, Perotti L, Kim AJ, Fenk LM, Cheng C, Maimon G, Zhao C, Widmer Y, Sprecher S, Senn W, Halnes G, Mäki-Marttunen T, Keller D, Pettersen KH, Andreassen OA, Einevoll GT, Yamada Y, Steyn-Ross ML, Alistair Steyn-Ross D, Mejias JF, Murray JD, Kennedy H, Wang XJ, Kruscha A, Grewe J, Benda J, Lindner B, Badel L, Ohta K, Tsuchimoto Y, Kazama H, Kahng B, Tam ND, Pollonini L, Zouridakis G, Soh J, Kim D, Yoo M, Palmer SE, Culmone V, Bojak I, Ferrario A, Merrison-Hort R, Borisyuk R, Kim CS, Tezuka T, Joo P, Rho YA, Burt… See abstract for full author list ➔ Sharpee TO, et al. BMC Neurosci. 2016 Aug 18;17 Suppl 1(Suppl 1):54. doi: 10.1186/s12868-016-0283-6. BMC Neurosci. 2016. PMID: 27534393 Free PMC article.
Jirsa P130 Epileptic seizures in the unfolding of a codimension-3 singularity Maria L. Saggio, Silvan F. Siep, Andreas Spiegler, William C. Stacey, Christophe Bernard, Viktor K. ...Graham P154 Quantifying resilience patterns in brain networks: the importance of directional …
Jirsa P130 Epileptic seizures in the unfolding of a codimension-3 singularity Maria L. Saggio, Silvan F. Siep, Andreas Spiegler, Will …
109 results