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407 results

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Page 1
Somatic reversion of pathogenic DOCK8 variants alters lymphocyte differentiation and function to effectively cure DOCK8 deficiency.
Pillay BA, Fusaro M, Gray PE, Statham AL, Burnett L, Bezrodnik L, Kane A, Tong W, Abdo C, Winter S, Chevalier S, Levy R, Masson C, Schmitt Y, Bole C, Malphettes M, Macintyre E, De Villartay JP, Ziegler JB, Smart JM, Peake J, Aghamohammadi A, Hammarström L, Abolhassani H, Picard C, Fischer A, Latour S, Neven B, Tangye SG, Ma CS. Pillay BA, et al. Among authors: macintyre e. J Clin Invest. 2021 Feb 1;131(3):e142434. doi: 10.1172/JCI142434. J Clin Invest. 2021. PMID: 33290277 Free PMC article. Clinical Trial.
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.
Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asnafi V, MacIntyre E, Dal Cortivo L, Radford I, Brousse N, Sigaux F, Moshous D, Hauer J, Borkhardt A, Belohradsky BH, Wintergerst U, Velez MC, Leiva L, Sorensen R, Wulffraat N, Blanche S, Bushman FD, Fischer A, Cavazzana-Calvo M. Hacein-Bey-Abina S, et al. Among authors: macintyre e. J Clin Invest. 2008 Sep;118(9):3132-42. doi: 10.1172/JCI35700. J Clin Invest. 2008. PMID: 18688285 Free PMC article. Clinical Trial.
Occurrence of myelodysplastic syndrome in 2 patients with reticular dysgenesis.
Lagresle-Peyrou C, Neven B, Six E, Picard C, Demerens-de Chappedelaine C, Bertrand Y, Jabado N, Chomienne C, Radford-Weiss I, Brouzes C, Asnafi V, MacIntyre E, Donadieu J, Beaupain B, Fenaux P, Eclache V, Fischer A, Cavazzana-Calvo M. Lagresle-Peyrou C, et al. Among authors: macintyre e. J Allergy Clin Immunol. 2011 Jul;128(1):230-232.e2. doi: 10.1016/j.jaci.2011.02.008. Epub 2011 Mar 31. J Allergy Clin Immunol. 2011. PMID: 21458044 No abstract available.
Loss of ARHGEF1 causes a human primary antibody deficiency.
Bouafia A, Lofek S, Bruneau J, Chentout L, Lamrini H, Trinquand A, Deau MC, Heurtier L, Meignin V, Picard C, Macintyre E, Alibeu O, Bras M, Molina TJ, Cavazzana M, André-Schmutz I, Durandy A, Fischer A, Oksenhendler E, Kracker S. Bouafia A, et al. Among authors: macintyre e. J Clin Invest. 2019 Mar 1;129(3):1047-1060. doi: 10.1172/JCI120572. Epub 2019 Feb 4. J Clin Invest. 2019. PMID: 30521495 Free PMC article.
Safety and efficacy of brentuximab vedotin as a treatment for lymphoproliferative disorders in primary immunodeficiencies.
Pincez T, Bruneau J, Berteloot L, Piekarski E, Thomas C, Marçais A, Trinquand A, Castelle M, Garcelon N, Plantaz D, Cheminant M, Moshous D, Molina TJ, Hermine O, Macintyre E, Fischer A, Blanche S, Suarez F, Neven B. Pincez T, et al. Among authors: macintyre e. Haematologica. 2020 Sep 1;105(9):e461-464. doi: 10.3324/haematol.2019.230276. Haematologica. 2020. PMID: 33054064 Free PMC article. No abstract available.
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome.
Magnani A, Semeraro M, Adam F, Booth C, Dupré L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Magnani A, et al. Among authors: macintyre e. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24. Nat Med. 2022. PMID: 35075289 Free PMC article.
Human kidney-derived hematopoietic stem cells can support long-term multilineage hematopoiesis.
Sobrino S, Abdo C, Neven B, Denis A, Gouge-Biebuyck N, Clave E, Charbonnier S, Blein T, Kergaravat C, Alcantara M, Villarese P, Berthaud R, Dehoux L, Albinni S, Karkeni E, Lagresle-Peyrou C, Cavazzana M, Salomon R, André I, Toubert A, Asnafi V, Picard C, Blanche S, Macintyre E, Boyer O, Six E, Zuber J. Sobrino S, et al. Among authors: macintyre e. Kidney Int. 2023 Jan;103(1):70-76. doi: 10.1016/j.kint.2022.08.024. Epub 2022 Sep 13. Kidney Int. 2023. PMID: 36108807
407 results