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Karen Buckland

university college london
H指数:17
被引数:2,385

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Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy

使用造血干细胞基因疗法对神经退行性黏多糖贮积症IIIA患者进行临床和生化纠正

2026-09-250
PRE
Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease

临床前体外IL2RG基因治疗,使用自体造血干细胞作为X连锁重症联合免疫缺陷病的一种有效且安全的治疗方法

2025-05-010
OAAI
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

自体慢病毒基因治疗腺苷脱氨酶缺乏症

2021-05-27131
OA
Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial
2021-02-012
PREAI
Lentiviral gene therapy for X-linked chronic granulomatous disease
2020-01-27169
OAAI
Outcomes Following Gene Therapy in Patients With Severe Wiskott-Aldrich Syndrome
2015-04-21309
OAAI
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
2014-10-09328
OAAI