Towards Clinical Translation of Hematopoietic Cell Gene Editing for Treating Hyper-IgM Type 1

被引：0
|
作者：
Vavassori, Valentina [1 ,2 ]
Mercuri, Elisabetta [1 ]
Marcovecchio, Genni [1 ]
Castiello, Maria Carmina [1 ,3 ]
Canarutto, Daniele [1 ,2 ,4 ]
Asperti, Claudia [1 ]
Jacob, Aurelien [1 ]
Albano, Luisa [1 ]
Fontana, Elena [5 ]
Scanziani, Eugenio [5 ]
Radrizzani, Marina [1 ,3 ]
Villa, Anna [1 ]
Genovese, Pietro [6 ]
Naldini, Luigi [1 ,2 ]
机构：
[1] IRCCS San Raffaele Sci Inst, San Raffaele Telethon Inst Gene Therapy, Milan, Italy
[2] Univ Vita Salute San Raffaele, Milan, Italy
[3] CNR, Milan, Italy
[4] IRCCS San Raffaele Sci Inst, Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy
[5] Humanitas, Milan, Italy
[6] Boston Childrens Hosp, Boston, MA USA
来源：
BLOOD | 2021年 / 138卷
关键词：
D O I：
10.1182/blood-2021-148572
中图分类号：
R5 [内科学];
学科分类号：
1002 ; 100201 ;
摘要：
3978
引用
收藏
页数：3
相关论文
共 50 条
  • [21] Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation
    Ferrari, Samuele
    Vavassori, Valentina
    Canarutto, Daniele
    Jacob, Aurelien
    Castiello, Maria Carmina
    Javed, Attya Omer
    Genovese, Pietro
    FRONTIERS IN GENOME EDITING, 2021, 3
  • [22] Safety assessment of gene edited CD4+ T-cells for the treatment of Hyper-IgM 1
    Canarutto, D.
    Vavassori, V.
    Ferrari, S.
    Plati, T.
    Porcellini, S.
    Asperti, C.
    Radrizzani, M.
    Paulis, M.
    Villa, A.
    Naldini, L.
    HUMAN GENE THERAPY, 2022, 33 (23-24) : A107 - A108
  • [23] Towards The Clinical Translation of Gene Correction in Hematopoietic Stem Cells for Sickle Cell Disease Treatment
    Lattanzi, Annalisa
    Dever, Daniel P.
    Camarena, Joab
    Lahiri, Premanjali
    Segal, Helen
    Talbott, Narae
    Srifa, Wai
    Cromer, Kyle
    Lee, Ciaran
    Bao, Gang
    Bathia, Neehar
    Uchida, Naoya
    Tisdale, John F.
    Porteus, Matthew H.
    MOLECULAR THERAPY, 2019, 27 (04) : 448 - 448
  • [24] Towards the clinical translation of stem cell therapy for type 1 diabetes
    Espes, Daniel
    Lau, Joey
    Carlsson, Per-Ola
    EUROPEAN JOURNAL OF ENDOCRINOLOGY, 2017, 177 (04) : R159 - R168
  • [25] ARDS as Presenting Symptom in an Infant with CD40L Deficiency (Hyper-IgM Syndrome Type 1)
    Fremerey, C.
    Wiebe, B.
    Feyen, O.
    Lenski, C.
    Pohlmann, U.
    Ehlen, M.
    Schofer, O.
    Meindl, A.
    Niehuess, T.
    Bartmann, P.
    KLINISCHE PADIATRIE, 2009, 221 (05): : 302 - 304
  • [26] Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation
    de la Morena, M. Teresa
    Leonard, David
    Torgerson, Troy R.
    Cabral-Marques, Otavio
    Slatter, Mary
    Aghamohammadi, Asghar
    Chandra, Sharat
    Murguia-Favela, Luis
    Bonilla, Francisco A.
    Kanariou, Maria
    Damrongwatanasuk, Rongras
    Kuo, Caroline Y.
    Dvorak, Christopher C.
    Meyts, Isabelle
    Chen, Karin
    Kobrynski, Lisa
    Kapoor, Neena
    Richter, Darko
    DiGiovanni, Daniela
    Dhalla, Fatima
    Farmaki, Evangelia
    Speckmann, Carsten
    Espanol, Teresa
    Shcherbina, Anna
    Hanson, Imelda Celine
    Litzman, Jiri
    Routes, John M.
    Wong, Melanie
    Fuleihan, Ramsay
    Seneviratne, Suranjith L.
    Small, Trudy N.
    Janda, Ales
    Bezrodnik, Liliana
    Seger, Reinhard
    Raccio, Andrea Gomez
    Edgar, J. David M.
    Chou, Janet
    Abbott, Jordan K.
    van Montfrans, Joris
    Gonzalez-Granado, Luis Ignacio
    Bunin, Nancy
    Kutukculer, Necil
    Gray, Paul
    Seminario, Gisela
    Pasic, Srdjan
    Aquino, Victor
    Wysocki, Christian
    Abolhassani, Hassan
    Dorsey, Morna
    Cunningham-Rundles, Charlotte
    JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, 2017, 139 (04) : 1282 - 1292
  • [27] Hematopoietic stem cell transplant for hyper-IgM syndrome due to CD40L defects: A single-center experience
    Al-Saud, Bandar
    Al-Mousa, Hamoud
    Al-Ahmari, Ali
    Al-Ghonaium, Abdulaziz
    Ayas, Mouhab
    Alhissi, Safa
    Al-Muhsen, Saleh
    Al-Seraihy, Amal
    Arnaout, Rand
    Al-Dhekri, Hasan
    Hawwari, Abbas
    PEDIATRIC TRANSPLANTATION, 2015, 19 (06) : 634 - 639
  • [28] Commentary: CRISPR gene editing for inherited retinal dystrophies: Towards clinical translation
    Bansal, Mayank
    Chakraborty, Debojyoti
    INDIAN JOURNAL OF OPHTHALMOLOGY, 2022, 70 (07) : 2326 - 2327
  • [29] Development of Lentiviral Vectors for Gene Therapy of X-Linked Hyper-IGM Syndrome (X-HIGM1)
    Romero, Z.
    Cobo, M.
    Unciti, J. D.
    Martin, F.
    Molina, I.
    HUMAN GENE THERAPY, 2009, 20 (09) : 1043 - 1043
  • [30] ANALYSIS OF X-CHROMOSOME INACTIVATION IN X-LINKED IMMUNODEFICIENCY WITH HYPER-IGM (HIGM1) - EVIDENCE FOR INVOLVEMENT OF DIFFERENT HEMATOPOIETIC-CELL LINEAGES
    NOTARANGELO, LD
    PAROLINI, O
    ALBERTINI, A
    DUSE, M
    MAZZOLARI, E
    PLEBANI, A
    CAMERINO, G
    UGAZIO, AG
    HUMAN GENETICS, 1991, 88 (02) : 130 - 134
12345