Biblio

Author Title Type [ Year(Asc)]
Filters: Author is Malech, Harry L  [Clear All Filters]
2024
Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SSi, Deal CL, et al. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: a PIDTC study. J Allergy Clin Immunol. 2024.
Castiello MCarmina, Brandas C, Ferrari S, Porcellini S, Sacchetti N, Canarutto D, Draghici E, Merelli I, Barcella M, Pelosi G, et al. Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency. Sci Transl Med. 2024;16(733):eadh8162.
2023
Yang AH, Sullivan B, Zerbe CS, De Ravin SSee, Blakely AM, Quezado MM, Marciano BE, Marko J, Ling A, Kleiner DE, et al. Gastrointestinal and Hepatic Manifestations of Chronic Granulomatous Disease. J Allergy Clin Immunol Pract. 2023.
Pavel-Dinu M, Gardner CL, Nakauchi Y, Kawai T, Delmonte OM, Palterer B, Bosticardo M, Pala F, Viel S, Malech HL, et al. Genetically Corrected RAG2-SCID Human Hematopoietic Stem Cells Restore V(D)J-Recombinase and Rescue Lymphoid Deficiency. Blood Adv. 2023.
Tamaoki N, Siebert S, Maeda T, Ha N-H, Good ML, Huang Y, Vodnala SK, Haro-Mora JJ, Uchida N, Tisdale JF, et al. Self-organized yolk sac-like organoids allow for scalable generation of multipotent hematopoietic progenitor cells from induced pluripotent stem cells. Cell Rep Methods. 2023;3(4):100460.
2020
Lin CH, Kuehn HSun, Thauland TJ, Lee CM, De Ravin SSee, Malech HL, Keyes TJ, Jager A, Davis KL, Garcia-Lloret MI, et al. Progressive B Cell Loss in Revertant X-SCID. J Clin Immunol. 2020.
2019
Sweeney CL, Merling RK, De Ravin SSee, Choi U, Malech HL. Gene Editing in Chronic Granulomatous Disease. Methods Mol Biol. 2019;1982:623-665.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, et al. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med. 2019;380(16):1525-1534.
2016
Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta KG, et al. Lentivirus Mediated Correction of Artemis-deficient Severe Combined Immunodeficiency. Hum Gene Ther. 2016.
De Ravin SSee, Reik A, Liu P-Q, Li L, Wu X, Su L, Raley C, Theobald N, Choi U, Song AH, et al. Targeted gene addition in human CD34(+) hematopoietic cells for correction of X-linked chronic granulomatous disease. Nat Biotechnol. 2016.
2014
De Ravin SSee, Su L, Theobald N, Choi U, Macpherson JL, Poidinger M, Symonds G, Pond SM, Ferris AL, Hughes SH, et al. Enhancers Are Major Targets for MLV Vector Integration. J Virol. 2014.
McDermott DH, Liu Q, Velez D, Lopez L, Anaya-O'Brien S, Ulrick J, Kwatemaa N, Starling J, Fleisher TA, Priel DALong, et al. A phase 1 clinical trial of long-term, low-dose treatment of WHIM syndrome with the CXCR4 antagonist plerixafor. Blood. 2014.