Hiroshi Kobayashi1

  • 1Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, 3-5-28, Nishi-Shimbashi, Minato-ku, Tokyo 105-8461, Japan.

Brain & development
|August 9, 2025
PubMed
概括

基因疗法对溶酶体储存疾病 (LSDs) 有希望,但安全问题需要进一步研究. 了解这些代谢障碍是开发有效和安全治疗的关键.

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