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Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy.
von Drygalski A, Gomez E, Giermasz A, Castaman G, Key NS, Lattimore SU, Leebeek FWG, Miesbach WA, Recht M, Gut R, Dolmetsch R, Monahan PE, Le Quellec S, Pipe SW. von Drygalski A, et al. Among authors: dolmetsch r. Blood Adv. 2023 Oct 10;7(19):5671-5679. doi: 10.1182/bloodadvances.2022008886. Blood Adv. 2023. PMID: 36490302 Free PMC article.
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
Pipe SW, Leebeek FWG, Recht M, Key NS, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O'Connell N, Pasi KJ, Hart DP, Kazmi R, Astermark J, Hermans CRJR, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary SE, Escobar M, Gomez E, Kruse-Jarres R, Quon DV, Symington E, Wang M, Wheeler AP, Gut R, Liu YP, Dolmetsch RE, Cooper DL, Li Y, Goldstein B, Monahan PE. Pipe SW, et al. Among authors: dolmetsch re. N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644. N Engl J Med. 2023. PMID: 36812434 Clinical Trial.
Patient Brain Organoids Identify a Link between the 16p11.2 Copy Number Variant and the RBFOX1 Gene.
Kostic M, Raymond JJ, Freyre CAC, Henry B, Tumkaya T, Khlghatyan J, Dvornik J, Li J, Hsiao JS, Cheon SH, Chung J, Sun Y, Dolmetsch RE, Worringer KA, Ihry RJ. Kostic M, et al. Among authors: dolmetsch re. ACS Chem Neurosci. 2023 Nov 15;14(22):3993-4012. doi: 10.1021/acschemneuro.3c00442. Epub 2023 Oct 30. ACS Chem Neurosci. 2023. PMID: 37903506 Free PMC article.
The 22q11.2 region regulates presynaptic gene-products linked to schizophrenia.
Nehme R, Pietiläinen O, Artomov M, Tegtmeyer M, Valakh V, Lehtonen L, Bell C, Singh T, Trehan A, Sherwood J, Manning D, Peirent E, Malik R, Guss EJ, Hawes D, Beccard A, Bara AM, Hazelbaker DZ, Zuccaro E, Genovese G, Loboda AA, Neumann A, Lilliehook C, Kuismin O, Hamalainen E, Kurki M, Hultman CM, Kähler AK, Paulo JA, Ganna A, Madison J, Cohen B, McPhie D, Adolfsson R, Perlis R, Dolmetsch R, Farhi S, McCarroll S, Hyman S, Neale B, Barrett LE, Harper W, Palotie A, Daly M, Eggan K. Nehme R, et al. Among authors: dolmetsch r. Nat Commun. 2022 Jun 27;13(1):3690. doi: 10.1038/s41467-022-31436-8. Nat Commun. 2022. PMID: 35760976 Free PMC article.
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.
Sabatino DE, Bushman FD, Chandler RJ, Crystal RG, Davidson BL, Dolmetsch R, Eggan KC, Gao G, Gil-Farina I, Kay MA, McCarty DM, Montini E, Ndu A, Yuan J; American Society of Gene and Cell Therapy (ASGCT) Working Group on AAV Integration. Sabatino DE, et al. Among authors: dolmetsch r. Mol Ther. 2022 Aug 3;30(8):2646-2663. doi: 10.1016/j.ymthe.2022.06.004. Epub 2022 Jun 10. Mol Ther. 2022. PMID: 35690906 Free PMC article. Review.
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion.
Keller CG, Shin Y, Monteys AM, Renaud N, Beibel M, Teider N, Peters T, Faller T, St-Cyr S, Knehr J, Roma G, Reyes A, Hild M, Lukashev D, Theil D, Dales N, Cha JH, Borowsky B, Dolmetsch R, Davidson BL, Sivasankaran R. Keller CG, et al. Among authors: dolmetsch r. Nat Commun. 2022 Mar 3;13(1):1150. doi: 10.1038/s41467-022-28653-6. Nat Commun. 2022. PMID: 35241644 Free PMC article.
Genome-wide CRISPR screen identifies protein pathways modulating tau protein levels in neurons.
Sanchez CG, Acker CM, Gray A, Varadarajan M, Song C, Cochran NR, Paula S, Lindeman A, An S, McAllister G, Alford J, Reece-Hoyes J, Russ C, Craig L, Capre K, Doherty C, Hoffman GR, Luchansky SJ, Polydoro M, Dolmetsch R, Elwood F. Sanchez CG, et al. Among authors: dolmetsch r. Commun Biol. 2021 Jun 14;4(1):736. doi: 10.1038/s42003-021-02272-1. Commun Biol. 2021. PMID: 34127790 Free PMC article.
82 results