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Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
Río P, Zubicaray J, Navarro S, Gálvez E, Sánchez-Domínguez R, Nicoletti E, Sebastián E, Rothe M, Pujol R, Bogliolo M, John-Neek P, Bastone AL, Schambach A, Wang W, Schmidt M, Larcher L, Segovia JC, Yáñez RM, Alberquilla O, Díez B, Fernández-García M, García-García L, Ramírez M, Galy A, Lefrere F, Cavazzana M, Leblanc T, García de Andoin N, López-Almaraz R, Catalá A, Barquinero J, Rodríguez-Perales S, Rao G, Surrallés J, Soulier J, Díaz-de-Heredia C, Schwartz JD, Sevilla J, Bueren JA; FANCOLEN-1 gene therapy investigators. Río P, et al. Lancet. 2025 Dec 21;404(10471):2584-2592. doi: 10.1016/S0140-6736(24)01880-4. Epub 2024 Dec 3. Lancet. 2025. PMID: 39642902 Clinical Trial.
PUFA stabilizes a conductive state of the selectivity filter in IKs channels.
Golluscio A, Eldstrom J, Jowais JJ, Perez ME, Cunningham KP, De La Cruz A, Wu X, Corradi V, Tieleman DP, Fedida D, Larsson HP. Golluscio A, et al. Among authors: de la cruz a. Elife. 2024 Oct 31;13:RP95852. doi: 10.7554/eLife.95852. Elife. 2024. PMID: 39480699 Free PMC article.
Astrocytic GLUT1 reduction paradoxically improves central and peripheral glucose homeostasis.
Ardanaz CG, de la Cruz A, Minhas PS, Hernández-Martín N, Pozo MÁ, Valdecantos MP, Valverde ÁM, Villa-Valverde P, Elizalde-Horcada M, Puerta E, Ramírez MJ, Ortega JE, Urbiola A, Ederra C, Ariz M, Ortiz-de-Solórzano C, Fernández-Irigoyen J, Santamaría E, Karsenty G, Brüning JC, Solas M. Ardanaz CG, et al. Among authors: de la cruz a. Sci Adv. 2024 Oct 18;10(42):eadp1115. doi: 10.1126/sciadv.adp1115. Epub 2024 Oct 18. Sci Adv. 2024. PMID: 39423276 Free PMC article.
Lipophilic compounds restore function to neurodevelopmental-associated KCNQ3 mutations.
Edmond MA, Hinojo-Perez A, Efrem M, Yi-Chun L, Shams I, Hayoz S, de la Cruz A, Perez Rodriguez ME, Diaz-Solares M, Dykxhoorn DM, Luo YL, Barro-Soria R. Edmond MA, et al. Among authors: de la cruz a. Commun Biol. 2024 Sep 19;7(1):1181. doi: 10.1038/s42003-024-06873-4. Commun Biol. 2024. PMID: 39300259 Free PMC article.
290 results