造血干细胞基因治疗脑罹。
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为F,邓肯C, Musolino PL,果园PJ, De Oliveira年代,脱粒机AJ, Armant M, Dansereau C,隆德TC,米勒WP,雷蒙德•问Sankar R,沙AJ,西维因C,加斯帕HB, P,实现一定程度Amartino H, Bratkovic D,史密斯NJC,帕克,沙米尔E O ' meara T,戴维森D,奥堡P,威廉姆斯哒
造血干细胞基因治疗脑罹。
郑传经地中海J。2017年10月26日,377 (17):1630 - 1638。doi: 10.1056 / NEJMoa1700554。Epub 2017 10月4。
- PubMed ID
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28976817 (在PubMed]
- 文摘
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背景:在x连锁罹,在ABCD1基因突变导致肾上腺白质退化症患者蛋白质功能的丧失。脑罹特点是脱髓鞘和神经退化。疾病进展,从而导致神经功能的受损和死亡,只能暂停与同种异体造血干细胞移植。方法:我们招收男孩大脑罹单一群体,非盲、阶段2 - 3安全性和有效性的研究。患者需要早期疾病和钆增强磁共振成像(MRI)在筛选。所涉及的试验药物注入自体CD34 +细胞转导与elivaldogene tavalentivec (Lenti-D)慢病毒载体。在这个临时分析,患者评估对移植物抗宿主病的发生,死亡,和主要功能障碍,神经系统功能的变化以及在核磁共振成像病变的程度。主要终点是活着,没有主要功能残疾输液后24个月。结果:共有17个男孩收到Lenti-D基因治疗。时的临时分析,平均随访29.4个月(范围,21.6到42.0)。 All the patients had gene-marked cells after engraftment, with no evidence of preferential integration near known oncogenes or clonal outgrowth. Measurable ALD protein was observed in all the patients. No treatment-related death or graft-versus-host disease had been reported; 15 of the 17 patients (88%) were alive and free of major functional disability, with minimal clinical symptoms. One patient, who had had rapid neurologic deterioration, had died from disease progression. Another patient, who had had evidence of disease progression on MRI, had withdrawn from the study to undergo allogeneic stem-cell transplantation and later died from transplantation-related complications. CONCLUSIONS: Early results of this study suggest that Lenti-D gene therapy may be a safe and effective alternative to allogeneic stem-cell transplantation in boys with early-stage cerebral adrenoleukodystrophy. Additional follow-up is needed to fully assess the duration of response and long-term safety. (Funded by Bluebird Bio and others; STARBEAM ClinicalTrials.gov number, NCT01896102 ; ClinicalTrialsRegister.eu number, 2011-001953-10 .).
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