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H

Harry L. Malech

national institutes of health (nih) - usa
H指数:96
被引数:3.4万

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Additive effects of common and rare genetic variants on inflammatory bowel disease risk in a cohort with immunodeficiency

常见和稀有遗传变异对免疫缺陷队列中炎症性肠病风险的累加效应

2026-02-270
PRE
Cryptosporidiosis in Inborn Errors of Immunity: A Single Center Cohort and Review of the Literature

免疫缺陷病中的隐孢子虫病:一项单中心队列研究及文献综述

2026-01-010
PRE
Prime Editing for p47phox-Deficient Chronic Granulomatous Disease

Gori, J.L.; Haddad, E.; Frangoul, H.; Kohn, D.B.; Morris, E.C.; Martin, B.N.; Deary, B.A.; Nickerson, M.; Scholz, R.L.; Fernandez, I.; et al. 基因组编辑技术用于p47phox缺陷型慢性肉芽肿病。N. Engl. J. Med. 2025. [Google Scholar] [CrossRef] [PubMed]

2025-12-010
PRE
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency

腺苷脱氨酶缺乏症的基因治疗长期安全性与有效性

2025-10-160
PRE
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替代方案:一份PIDTC报告

2025-10-020
PRE
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)在异基因造血细胞移植后弓形虫病诊断与管理中的有效性与局限性:单中心16年经验,涉及31例患者

2025-08-050
OAAI
Platform solutions for commercial challenges to expanding patient access and making gene editing sustainable

针对商业挑战的平台解决方案,以扩大患者访问范围并使基因编辑可持续发展

2025-07-150
PRE
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改善罕见病治疗方案的可行性:2024年由ARM、ISCT和Danaher主办的科学交流会的关键见解

2025-06-240
PREAI
High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease

造血干细胞的高保真无PAMless碱基编辑治疗慢性肉芽肿病

2024-10-160
PRE
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A Primary Immune Deficiency Treatment Consortium study
2024-05-013
PRE
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency

基因校正的RAG2-SCID人造血干细胞恢复V(D)J-重组酶和拯救淋巴缺陷

2024-04-091
OAAI
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency
2024-02-075
OA
Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency Treatment Consortium (PIDTC) landmark study
2024-01-014
OAAI
Intestinal microbiome and metabolome signatures in patients with chronic granulomatous disease
2023-12-018
OAAI
A phase III randomized crossover trial of plerixafor versus G-CSF for treatment of WHIM syndrome
2023-10-0212
OAAI
Small bowel disease prevalence on video capsule endoscopy in chronic granulomatous disease-associated inflammatory bowel disease

视频胶囊内镜在慢性肉芽肿病相关炎症性肠病中的小肠疾病患病率

2023-09-242
OA
Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients
2023-06-016
OAAI