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The clinical protocol to treat infants with severe combined immuno deficiency due to adenosine deaminase (ADA) deficiency by means of gene thereapy has been approved recently. The Human Gene Therapy Subcommittee and the Recombinant DNA Advisory Committee (RAC) of the National Institutes of Health (NIH) concluded the review on July 30th and opened the door for the first federally approved somatic Human Gene Therapy.
一项通过基因疗法治疗因腺苷脱氨酶(ADA)缺乏所致严重联合免疫缺陷婴儿的临床方案最近已获批准。美国国立卫生研究院(NIH)的人类基因治疗小组委员会和重组DNA咨询委员会(RAC)于7月30日完成了审查,为首个获得联邦批准的体细胞人类基因治疗开启了大门。