佛州医生用基因疗法眼药水让盲人少年喜见光明
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佛州有医生试用基因治疗眼药水后,成功地令1名失明少年恢复视力,医学界相信这种技术潜力巨大,开启大门后,料可造福数以百万计眼疾患者。
美联社报道,接受治疗的14岁少年卡尔瓦哈尔(Antonio Vento
Carvajal)患有罕见的遗传疾病,其皮肤和黏膜组织非常脆弱,导致眼睛形成水泡和溃疡,幸而参加迈阿密大学帕尔默眼科研究所的治疗计划后,病情迎来了转机。研究所的医生萨巴特(Alfonso
Sabater)表示,较早前他参与同行的临床试验,使用局部基因药物治疗皮肤病,产生灵感后尝试把同样方法用于眼疾。
卡尔瓦哈尔于2012年持特殊签证,随家人从古巴来美求医,一度接受手术切除眼睛的疤痕组织,但不久后疤痕重新长出,导致他的视力不断恶化,最终日常行走也遇到困难。卡尔瓦哈尔的母亲表示,萨巴特医生不断鼓励他们不要放弃,因此家人始终抱持希望,医生得知基因治疗皮肤的技术后,亲自联系药物凝胶的制造商Krystal
Biotech,查询凝胶能否用于眼睛。
据悉,这种方法使用灭活的单纯
疹病毒,将基因拷贝到患者身上,随后能产生1种特别的胶原蛋白,修复皮肤和眼角膜。而在卡尔瓦哈尔的个案中,他使用的眼药水属于液体版本,当中没有添加凝胶,但配方和用于皮肤的药物相同。
为了这次创举,医生团队测试2年,获得大学、医院、联邦食品及药物管理局(FDA)许可后,去年8月首先治疗卡尔瓦哈尔的右眼,数月后发现眼睛再没有出现疤痕,各个月情况都在进步,最近测试更显示,卡尔瓦哈尔右眼的视力恢复到20/25。
萨巴特医生表示,今年会开始治疗卡尔瓦哈尔的左眼,左眼疤痕组织更多,治疗难度较大,但情况也正在稳步改善。
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