日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Umbilical Cord Blood Transplantation Provides an Alternative for Patients With Chronic Granulomatous Disease Lacking HLA-Matched Donors: A PIDTC Report

脐带血移植为缺乏HLA匹配供体的慢性肉芽肿病患者提供了一种替代方案:PIDTC报告

Arnold, Danielle E; Leiding, Jennifer W; Logan, Brent; Marsh, Rebecca A; Griffith, Linda M; Grunebaum, Eyal; Murguía-Favela, Luis; Mallhi, Kanwaldeep; Chellapandian, Deepakbabu; Deal, Christin L; Lim, Stephanie Si; Prasad, Vinod; Heimall, Jennifer; Chandrakasan, Shanmuganathan; Chen, Karin; Yu, Lolie C; Seroogy, Christine M; Gillio, Alfred; Bednarski, Jeffrey J; Kapoor, Neena; Moore, Theodore B; Cuvelier, Geoffrey D E; Touzot, Fabien; Rayes, Ahmad; Ebens, Christen L; Schaefer, Edo; Bauchat, Andrea; Chopek, Ashley; Burroughs, Lauri; Cowan, Morton J; Dvorak, Christopher C; Haddad, Elie; Kohn, Donald B; Notarangelo, Luigi D; Pai, Sung-Yun; Puck, Jennifer M; Pulsipher, Michael A; Torgerson, Troy; Malech, Harry L; Kang, Elizabeth M; Parikh, Suhag

Reliable genetic diagnosis of NCF1 (p47(phox))-deficient chronic granulomatous disease using high-throughput sequencing

利用高通量测序对NCF1(p47(phox))缺陷型慢性肉芽肿病进行可靠的基因诊断

Hsu, Amy P; Karlins, Eric; Lack, Justin; Pepper, T Joseph; Lau, Karen; Marshall-Batty, Kimberly R; Long Priel, Debra; Davis, Joie; Fink, Danielle L; Zerbe, Christa S; Gallin, John I; Malech, Harry L; Holland, Steven M; Kuhns, Douglas B

Outcomes in hematopoetic cell transplantation in the setting of mold infections in patients with chronic granulomatous disease

慢性肉芽肿病患者合并霉菌感染时造血干细胞移植的预后

Kline, Ahnika; Parta, Mark; Cuellar-Rodriguez, Jennifer; Gea-Banacloche, Juan; Kelly, Corin; Pittaluga, Stefania; Zerbe, Christa S; Holland, Steven M; Malech, Harry L; Kang, Elizabeth M

Usefulness and Limitations of Polymerase Chain Reaction (PCR) for the Diagnosis and Management of Toxoplasmosis Following Allogeneic Hematopoietic Cell Transplant: Single-center Experience With 31 Patients Over 16 Years

聚合酶链式反应(PCR)在同种异体造血干细胞移植后弓形虫病诊断和治疗中的应用价值及局限性:单中心16年31例患者的经验

Czech, Mary M; Jerussi, Theresa; Das, Sanchita; Lee, Rose; Kanakry, Christopher G; Kanakry, Jennifer; Dimitrova, Dimana; Hyder, Mustafa A; Rechache, Kamil; Trang, Winnie; Fowler, Daniel H; Bishop, Michael R; Childs, Richard W; Aue, Georg; Tisdale, John; Hsieh, Matthew; Fitzhugh, Courtney; Limerick, Emily; Hickstein, Dennis; Malech, Harry L; Kang, Elizabeth M; Pavletic, Steven; Arnold, Danielle E; Pai, Sung-Yun; Cuellar-Rodriguez, Jennifer; Gea-Banacloche, Juan C

Advancing gene-editing platforms to improve the viability of rare-disease therapeutics: key insights from a 2024 Scientific Exchange hosted by ARM, ISCT, and Danaher

推进基因编辑平台以提高罕见病疗法的可行性:由ARM、ISCT和丹纳赫公司联合主办的2024年科学交流会的主要见解

Urnov, Fyodor; Kassim, Sadik; Musunuru, Kiran; Liu, David; Lee, Ann; Barrera, Luis; Stetkiewicz, Pam; Bruno, Julianne; Hewitt, Matthew; Lister, Troy; Malech, Harry; Gasch, Lindsay; Diver, Matt; Gertler, Nicholas; Grignon, Felix; Le, Audrey; Lehmicke, Michael; Almendro-Navarro, Vanessa; Lembong, Josephine

High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease

利用高保真无PAM碱基编辑技术对造血干细胞进行治疗慢性肉芽肿病

Bzhilyanskaya, Vera; Ma, Linyuan; Liu, Siyuan; Fox, Lauren R; Whittaker, Madelynn N; Meis, Ronald J; Choi, Uimook; Lawson, Amanda; Ma, Michelle; Theobald, Narda; Burkett, Sandra; Sweeney, Colin L; Lazzarotto, Cicera R; Tsai, Shengdar Q; Lack, Justin B; Wu, Xiaolin; Dahl, Gary A; Malech, Harry L; Kleinstiver, Benjamin P; De Ravin, Suk See

Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study

移植后晚期并发症在重症联合免疫缺陷病(SCID)患者中随时间推移而增加:一项原发性免疫缺陷治疗联盟(PIDTC)里程碑式研究

Eissa, Hesham; Thakar, Monica S; Shah, Ami J; Logan, Brent R; Griffith, Linda M; Dong, Huaying; Parrott, Roberta E; O'Reilly, Richard J; Dara, Jasmeen; Kapoor, Neena; Forbes Satter, Lisa; Chandra, Sharat; Kapadia, Malika; Chandrakasan, Shanmuganathan; Knutsen, Alan; Jyonouchi, Soma C; Molinari, Lyndsay; Rayes, Ahmad; Ebens, Christen L; Teira, Pierre; Dávila Saldaña, Blachy J; Burroughs, Lauri M; Chaudhury, Sonali; Chellapandian, Deepak; Gillio, Alfred P; Goldman, Fredrick; Malech, Harry L; DeSantes, Kenneth; Cuvelier, Geoff D E; Rozmus, Jacob; Quinones, Ralph; Yu, Lolie C; Broglie, Larisa; Aquino, Victor; Shereck, Evan; Moore, Theodore B; Vander Lugt, Mark T; Mousallem, Talal I; Oved, Joeseph H; Dorsey, Morna; Abdel-Azim, Hisham; Martinez, Caridad; Bleesing, Jacob H; Prockop, Susan; Kohn, Donald B; Bednarski, Jeffrey J; Leiding, Jennifer; Marsh, Rebecca A; Torgerson, Troy; Notarangelo, Luigi D; Pai, Sung-Yun; Pulsipher, Michael A; Puck, Jennifer M; Dvorak, Christopher C; Haddad, Elie; Buckley, Rebecca H; Cowan, Morton J; Heimall, Jennifer

Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study

异基因造血干细胞移植对p47phox慢性肉芽肿病有效:一项原发性免疫缺陷治疗联盟研究

Grunebaum, Eyal; Arnold, Danielle E; Logan, Brent; Parikh, Suhag; Marsh, Rebecca A; Griffith, Linda M; Mallhi, Kanwaldeep; Chellapandian, Deepak; Lim, Stephanie Si; Deal, Christin L; Kapoor, Neena; Murguía-Favela, Luis; Falcone, Emilia Liana; Prasad, Vinod K; Touzot, Fabien; Bleesing, Jack J; Chandrakasan, Shanmuganathan; Heimall, Jennifer R; Bednarski, Jeffrey J; Broglie, Larisa A; Chong, Hey Jin; Kapadia, Malika; Prockop, Susan; Dávila Saldaña, Blachy J; Schaefer, Edo; Bauchat, Andrea L; Teira, Pierre; Chandra, Sharat; Parta, Mark; Cowan, Morton J; Dvorak, Christopher C; Haddad, Elie; Kohn, Donald B; Notarangelo, Luigi D; Pai, Sung-Yun; Puck, Jennifer M; Pulsipher, Michael A; Torgerson, Troy R; Malech, Harry L; Kang, Elizabeth M; Leiding, Jennifer W

Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

基因校正的RAG2-SCID人类造血干细胞可恢复V(D)J重组酶并挽救淋巴细胞缺陷

Pavel-Dinu, Mara; Gardner, Cameron L; Nakauchi, Yusuke; Kawai, Tomoki; Delmonte, Ottavia M; Palterer, Boaz; Bosticardo, Marita; Pala, Francesca; Viel, Sebastien; Malech, Harry L; Ghanim, Hana Y; Bode, Nicole M; Kurgan, Gavin L; Detweiler, Angela M; Vakulskas, Christopher A; Neff, Norma F; Sheikali, Adam; Menezes, Sherah T; Chrobok, Jade; Hernández González, Elaine M; Majeti, Ravindra; Notarangelo, Luigi D; Porteus, Matthew H

High symptom burden in female X-linked chronic granulomatous disease carriers

女性X连锁慢性肉芽肿病携带者症状负担重

Miranda, Mary Ann; Tsalatsanis, Athanasios; Trotter, Jessica R; Arnold, Danielle E; Squire, Jacqueline D; Kidd, Sharon; Parikh, Suhag; Marsh, Rebecca A; Griffith, Linda M; Mallhi, Kanwaldeep; Chellapandian, Deepak; Lim, Stephanie Si; Grunebaum, Eyal; Sullivan, Kathleen E; Newburger, Peter E; Dinauer, Mary C; Cowan, Morton J; Dvorak, Christopher C; Haddad, Elie; Kohn, Donald B; Notarangelo, Luigi D; Pai, Sung-Yun; Puck, Jennifer M; Pulsipher, Michael A; Torgerson, Troy R; Malech, Harry L; Kang, Elizabeth M; Morton, Felicia B; Leiding, Jennifer W