Biblio

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Journal Article
Khatri D, Putoux A, Cologne A, Kaltenbach S, Besson A, Bertiaux E, Guguin J, Fendler A, Dupont MA, Benoit-Pilven C, et al. Deficiency of the minor spliceosome component U4atac snRNA secondarily results in ciliary defects in human and zebrafish. Proc Natl Acad Sci U S A. 2023;120(9):e2102569120.
Petit A, Neven B, Min V, Mahlaoui N, Moshous D, Castelle M, Allouche M, Stérin A, Visentin S, Saultier P, et al. Impact of graft function on health status and quality of life in 112 very long-term survivors who received an HSCT for Inborn Errors of Immunity, a prospective study of the CEREDIH. Transplant Cell Ther. 2023.
Fischer M, Olbrich P, Hadjadj J, Aumann V, Bakhtiar S, Barlogis V, von Bismarck P, Bloomfield MPhDMarké, Booth C, Buddingh EP, et al. JAK-inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID and EBMT IEWP retrospective study. J Allergy Clin Immunol. 2023.
César T, Le MP, Klifa R, Castelle M, Fournier B, Levy R, Chbihi M, Courteille V, Moshous D, Blanche S, et al. Letermovir for CMV Prophylaxis in Very High-Risk Pediatric Hematopoietic Stem Cell Transplantation Recipients for Inborn Errors of Immunity. J Clin Immunol. 2023;44(1):6.
Gardin A, Castelle M, Pichard S, Cano A, Chabrol B, Piarroux J, Roubertie A, Nadjar Y, Guemann A-S, Tardieu M, et al. Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H: a retrospective study of 51 patients. Bone Marrow Transplant. 2022.
Leiding JW, Vogel TP, Santarlas VGJ, Mhaskar R, Smith MR, Carisey A, Vargas-Hernández A, Silva-Carmona M, Heeg M, Rensing-Ehl A, et al. Monogenic Early-Onset Lymphoproliferation and Autoimmunity: The Natural History of STAT3 GOF Syndrome. J Allergy Clin Immunol. 2022.
Missontsa MMillin, Bernaudin F, Fortin A, Dhedin N, Pondarré C, Yakouben K, Neven B, Castelle M, Cavazzana M, Lezeau H, et al. Ovarian tissue cryopreservation for fertility preservation before hematopoietic stem cell transplantation in patients with sickle cell disease: safety, ovarian function follow-up, and results of ovarian tissue transplantation. J Assist Reprod Genet. 2024.
Rossi M, Szepetowski S, Yakouben K, Paillard C, Sirvent A, Castelle M, Pegon C, Piguet C, Grain A, Angoso M, et al. Recent results of hematopoietic stem cell transplantation for thalassemia with busulfan-based conditioning regimen in France: improved thalassemia free survival despite frequent mixed chimerism. A retrospective study from the Francophone Society of Stem C. Bone Marrow Transplant. 2023.