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Four-Month Rifapentine Regimens with or without Moxifloxacin for Tuberculosis.

Dorman SE, Nahid P, Kurbatova EV, Phillips PPJ, Bryant K, Dooley KE, Engle M, Goldberg SV, Phan HTT, Hakim J, Johnson JL, Lourens M, Martinson NA, Muzanyi G, Narunsky K, Nerette S, Nguyen NV, Pham TH, Pierre S, Purfield AE, Samaneka W, Savic RM, Sanne I, Scott NA, Shenje J, Sizemore E, Vernon A, Waja Z, Weiner M, Swindells S, Chaisson RE; AIDS Clinical Trials Group; Tuberculosis Trials Consortium. Dorman SE, et al. N Engl J Med. 2021 May 6;384(18):1705-1718. doi: 10.1056/NEJMoa2033400. N Engl J Med. 2021. PMID: 33951360 Free PMC article. Clinical Trial.

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Two phase 3 trials of bapineuzumab in mild-to-moderate Alzheimer's disease.

Salloway S, Sperling R, Fox NC, Blennow K, Klunk W, Raskind M, Sabbagh M, Honig LS, Porsteinsson AP, Ferris S, Reichert M, Ketter N, Nejadnik B, Guenzler V, Miloslavsky M, Wang D, Lu Y, Lull J, Tudor IC, Liu E, Grundman M, Yuen E, Black R, Brashear HR; Bapineuzumab 301 and 302 Clinical Trial Investigators. Salloway S, et al. N Engl J Med. 2014 Jan 23;370(4):322-33. doi: 10.1056/NEJMoa1304839. N Engl J Med. 2014. PMID: 24450891 Free PMC article. Clinical Trial.

BACKGROUND: Bapineuzumab, a humanized anti-amyloid-beta monoclonal antibody, is in clinical development for the treatment of Alzheimer's disease. METHODS: We conducted two double-blind, randomized, placebo-controlled, phase 3 trials involving patients with mild-to-moderate …

BACKGROUND: Bapineuzumab, a humanized anti-amyloid-beta monoclonal antibody, is in clinical development for the treatment of Alzheimer's …

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Mometasone or Tiotropium in Mild Asthma with a Low Sputum Eosinophil Level.

Lazarus SC, Krishnan JA, King TS, Lang JE, Blake KV, Covar R, Lugogo N, Wenzel S, Chinchilli VM, Mauger DT, Dyer AM, Boushey HA, Fahy JV, Woodruff PG, Bacharier LB, Cabana MD, Cardet JC, Castro M, Chmiel J, Denlinger L, DiMango E, Fitzpatrick AM, Gentile D, Hastie A, Holguin F, Israel E, Jackson D, Kraft M, LaForce C, Lemanske RF Jr, Martinez FD, Moore W, Morgan WJ, Moy JN, Myers R, Peters SP, Phipatanakul W, Pongracic JA, Que L, Ross K, Smith L, Szefler SJ, Wechsler ME, Sorkness CA; National Heart, Lung, and Blood Institute AsthmaNet. Lazarus SC, et al. N Engl J Med. 2019 May 23;380(21):2009-2019. doi: 10.1056/NEJMoa1814917. Epub 2019 May 19. N Engl J Med. 2019. PMID: 31112384 Free PMC article. Clinical Trial.

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A super-enhancer-regulated RNA-binding protein cascade drives pancreatic cancer.

Antal CE, Oh TG, Aigner S, Luo EC, Yee BA, Campos T, Tiriac H, Rothamel KL, Cheng Z, Jiao H, Wang A, Hah N, Lenkiewicz E, Lumibao JC, Truitt ML, Estepa G, Banayo E, Bashi S, Esparza E, Munoz RM, Diedrich JK, Sodir NM, Mueller JR, Fraser CR, Borazanci E, Propper D, Von Hoff DD, Liddle C, Yu RT, Atkins AR, Han H, Lowy AM, Barrett MT, Engle DD, Evan GI, Yeo GW, Downes M, Evans RM. Antal CE, et al. Nat Commun. 2023 Sep 6;14(1):5195. doi: 10.1038/s41467-023-40798-6. Nat Commun. 2023. PMID: 37673892 Free PMC article.

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A cell type-aware framework for nominating non-coding variants in Mendelian regulatory disorders.

Lee AS, Ayers LJ, Kosicki M, Chan WM, Fozo LN, Pratt BM, Collins TE, Zhao B, Rose MF, Sanchis-Juan A, Fu JM, Wong I, Zhao X, Tenney AP, Lee C, Laricchia KM, Barry BJ, Bradford VR, Jurgens JA, England EM, Lek M, MacArthur DG, Lee EA, Talkowski ME, Brand H, Pennacchio LA, Engle EC. Lee AS, et al. Nat Commun. 2024 Sep 27;15(1):8268. doi: 10.1038/s41467-024-52463-7. Nat Commun. 2024. PMID: 39333082 Free PMC article.

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MACF1 Mutations Encoding Highly Conserved Zinc-Binding Residues of the GAR Domain Cause Defects in Neuronal Migration and Axon Guidance.

Dobyns WB, Aldinger KA, Ishak GE, Mirzaa GM, Timms AE, Grout ME, Dremmen MHG, Schot R, Vandervore L, van Slegtenhorst MA, Wilke M, Kasteleijn E, Lee AS, Barry BJ, Chao KR, Szczałuba K, Kobori J, Hanson-Kahn A, Bernstein JA, Carr L, D'Arco F, Miyana K, Okazaki T, Saito Y, Sasaki M, Das S, Wheeler MM, Bamshad MJ, Nickerson DA; University of Washington Center for Mendelian Genomics; Center for Mendelian Genomics at the Broad Institute of MIT and Harvard; Engle EC, Verheijen FW, Doherty D, Mancini GMS. Dobyns WB, et al. Am J Hum Genet. 2018 Dec 6;103(6):1009-1021. doi: 10.1016/j.ajhg.2018.10.019. Epub 2018 Nov 21. Am J Hum Genet. 2018. PMID: 30471716 Free PMC article.

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Cilia signaling and obesity.

Engle SE, Bansal R, Antonellis PJ, Berbari NF. Engle SE, et al. Semin Cell Dev Biol. 2021 Feb;110:43-50. doi: 10.1016/j.semcdb.2020.05.006. Epub 2020 May 25. Semin Cell Dev Biol. 2021. PMID: 32466971 Free PMC article. Review.

We discuss how neuronal cilia impact food intake potentially through leptin signaling and changes in ciliary G protein-coupled receptor (GPCR) signaling. We highlight several recent studies that have implicated the potential for cilia in peripheral tissues such as adipose …

We discuss how neuronal cilia impact food intake potentially through leptin signaling and changes in ciliary G protein-coupled recept …

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ImmunoPET Imaging of Trop2 Expression in Bladder Cancer Using [⁶⁴Cu]Cu-NOTA-Trodelvy.

Huang W, Wang T, Chao F, Yang Q, Mixdorf JC, Li L, Engle JW, Fan Y, Kang L, Cai W. Huang W, et al. Mol Pharm. 2025 Apr 7;22(4):2266-2275. doi: 10.1021/acs.molpharmaceut.5c00069. Epub 2025 Mar 9. Mol Pharm. 2025. PMID: 40059341

In HT-1376 models, [(64)Cu]Cu-NOTA-Trodelvy effectively visualized tumor as early as 12 h p.i. (10.30 1.45% ID/g), with tumor uptake increasing and peaking at 48 h p.i. (13.73 1.16% ID/g), highlighting its potential for tumor imaging. Control groups also demonstrate …

In HT-1376 models, [(64)Cu]Cu-NOTA-Trodelvy effectively visualized tumor as early as 12 h p.i. (10.30 1.45% ID/g), with tumor uptake …

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Noncoding variants alter GATA2 expression in rhombomere 4 motor neurons and cause dominant hereditary congenital facial paresis.

Tenney AP, Di Gioia SA, Webb BD, Chan WM, de Boer E, Garnai SJ, Barry BJ, Ray T, Kosicki M, Robson CD, Zhang Z, Collins TE, Gelber A, Pratt BM, Fujiwara Y, Varshney A, Lek M, Warburton PE, Van Ryzin C, Lehky TJ, Zalewski C, King KA, Brewer CC, Thurm A, Snow J, Facio FM, Narisu N, Bonnycastle LL, Swift A, Chines PS, Bell JL, Mohan S, Whitman MC, Staffieri SE, Elder JE, Demer JL, Torres A, Rachid E, Al-Haddad C, Boustany RM, Mackey DA, Brady AF, Fenollar-Cortés M, Fradin M, Kleefstra T, Padberg GW, Raskin S, Sato MT, Orkin SH, Parker SCJ, Hadlock TA, Vissers LELM, van Bokhoven H, Jabs EW, Collins FS, Pennacchio LA, Manoli I, Engle EC. Tenney AP, et al. Nat Genet. 2023 Jul;55(7):1149-1163. doi: 10.1038/s41588-023-01424-9. Epub 2023 Jun 29. Nat Genet. 2023. PMID: 37386251 Free PMC article.

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Gemogenovatucel-T (Vigil): bi-shRNA plasmid-based targeted immunotherapy.

Nemunaitis J, Stanbery L, Walter A, Wallraven G, Nemunaitis A, Horvath S, Bognar E, Rao D, Engle S, Brun S, Ghisoli M, Rocconi RP, Monk BJ, Coleman RL. Nemunaitis J, et al. Future Oncol. 2024;20(29):2149-2164. doi: 10.1080/14796694.2024.2376518. Epub 2024 Aug 5. Future Oncol. 2024. PMID: 39101448 Free PMC article. Review.

Construction of Vigil involves harvest of autologous malignant tissue for neoantigen targeting (ideally containing clonal neoantigens) followed by a two-day process involving transfection with a plasmid to provide a permissive 'training environment' for the patient's immun …

Construction of Vigil involves harvest of autologous malignant tissue for neoantigen targeting (ideally containing clonal neoantigens) follo …

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