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1987 1
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60 results

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Page 1
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.
Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kühl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Duncan CN, et al. Among authors: kuhl js. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541. N Engl J Med. 2024. PMID: 39383458 Free PMC article. Clinical Trial.
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kühl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Eichler F, et al. Among authors: kuhl js. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442. N Engl J Med. 2024. PMID: 39383459 Free PMC article. Clinical Trial.
Human CARMIL2 deficiency underlies a broader immunological and clinical phenotype than CD28 deficiency.
Lévy R, Gothe F, Momenilandi M, Magg T, Materna M, Peters P, Raedler J, Philippot Q, Rack-Hoch AL, Langlais D, Bourgey M, Lanz AL, Ogishi M, Rosain J, Martin E, Latour S, Vladikine N, Distefano M, Khan T, Rapaport F, Schulz MS, Holzer U, Fasth A, Sogkas G, Speckmann C, Troilo A, Bigley V, Roppelt A, Dinur-Schejter Y, Toker O, Bronken Martinsen KH, Sherkat R, Somekh I, Somech R, Shouval DS, Kühl JS, Ip W, McDermott EM, Cliffe L, Ozen A, Baris S, Rangarajan HG, Jouanguy E, Puel A, Bustamante J, Alyanakian MA, Fusaro M, Wang Y, Kong XF, Cobat A, Boutboul D, Castelle M, Aguilar C, Hermine O, Cheminant M, Suarez F, Yildiran A, Bousfiha A, Al-Mousa H, Alsohime F, Cagdas D, Abraham RS, Knutsen AP, Fevang B, Bhattad S, Kiykim A, Erman B, Arikoglu T, Unal E, Kumar A, Geier CB, Baumann U, Neven B; CARMIL2 Consortium; Rohlfs M, Walz C, Abel L, Malissen B, Marr N, Klein C, Casanova JL, Hauck F, Béziat V. Lévy R, et al. Among authors: kuhl js. J Exp Med. 2023 Feb 6;220(2):e20220275. doi: 10.1084/jem.20220275. Epub 2022 Dec 14. J Exp Med. 2023. PMID: 36515678 Free PMC article.
Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy.
Weinhofer I, Rommer P, Gleiss A, Ponleitner M, Zierfuss B, Waidhofer-Söllner P, Fourcade S, Grabmeier-Pfistershammer K, Reinert MC, Göpfert J, Heine A, Yska HAF, Casasnovas C, Cantarín V, Bergner CG, Mallack E, Forss-Petter S, Aubourg P, Bley A, Engelen M, Eichler F, Lund TC, Pujol A, Köhler W, Kühl JS, Berger J. Weinhofer I, et al. Among authors: kuhl js. EBioMedicine. 2023 Oct;96:104781. doi: 10.1016/j.ebiom.2023.104781. Epub 2023 Sep 7. EBioMedicine. 2023. PMID: 37683329 Free PMC article.
Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency: an EBMT-IEWP retrospective study.
Herrmann US, Felber M, Worth AJ, Haskologlu S, Dogu F, Lewis VA, Strahm B, Groll AH, Gennery AR, Hauck F, Wynn R, Coussons M, Meyts I, Lindemans CA, Bordon Cueto de Braem MV, Bredius RG, Kühl JS, Völler M, Zirngibl F, Zaidman I, Laberko A, Zeilhofer U, Hauri-Hohl MM, Lankester AC, Ikinciogullari A, Guilcher GMT, Hackenberg A, Yeşilipek MA, Davies EG, Hershfield M, Parikh SH, Gilbert P, Bettoni da Cunha Riehm C, Albert MH, Schulz AS, Honig M, Neven B, Güngör T. Herrmann US, et al. Among authors: kuhl js. Blood. 2025 Sep 22:blood.2025029640. doi: 10.1182/blood.2025029640. Online ahead of print. Blood. 2025. PMID: 40983033
Approaches to Invasive Fungal Diseases in Paediatric Cancer Centres: An Analysis of Current Practices and Challenges in Germany, Austria and Switzerland.
Seidel D, Pana ZD, Ebrahimi-Fakhari D, Butzer SK, Mehler K, Reinhold I, Simon A, Dohna-Schwake C, Mack I, Bodmer N, Niehues T, Claviez A, Längler A, Leipold A, Prokop A, Brummel B, Winkler B, Gruhn B, Classen CF, Friedrich C, Koenig C, Flotho C, Poyer F, Schilling F, Calaminus G, Sieben G, Schwabe GC, Reinhard H, Teltschik HM, Hengartner H, Stursberg J, Greiner J, Greil J, Leyh J, Kühl JS, Ehlert K, Bochennek K, Rohde M, Demmert M, Stiefel M, Eyrich M, Siepermann M, Frühwald M, Döring M, Nathrath M, Minkov M, Streiter M, Jones N, Naumann-Bartsch N, Jorch N, Beck O, Beier R, Crazzolara R, Kietz S, Vieth S, Fröhling S, Lobitz S, Ghosh S, Vallée TC, Müller T, Wiesel T, Däbritz T, Kontny U, Thiel U, Strenger V, Eberl WR, Cornely OA, Groll AH, Lehrnbecher T. Seidel D, et al. Among authors: kuhl js. Mycoses. 2025 Jun;68(6):e70074. doi: 10.1111/myc.70074. Mycoses. 2025. PMID: 40515497 Free PMC article.
Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents.
Wlodarski MW, Hirabayashi S, Pastor V, Starý J, Hasle H, Masetti R, Dworzak M, Schmugge M, van den Heuvel-Eibrink M, Ussowicz M, De Moerloose B, Catala A, Smith OP, Sedlacek P, Lankester AC, Zecca M, Bordon V, Matthes-Martin S, Abrahamsson J, Kühl JS, Sykora KW, Albert MH, Przychodzien B, Maciejewski JP, Schwarz S, Göhring G, Schlegelberger B, Cseh A, Noellke P, Yoshimi A, Locatelli F, Baumann I, Strahm B, Niemeyer CM; EWOG-MDS. Wlodarski MW, et al. Among authors: kuhl js. Blood. 2016 Mar 17;127(11):1387-97; quiz 1518. doi: 10.1182/blood-2015-09-669937. Epub 2015 Dec 23. Blood. 2016. PMID: 26702063 Free article.
60 results