皮肤的核酸疗法
Nucleic Acid Therapy for the Skin.
作者信息
Chai Andreas C, Siegwart Daniel J, Wang Richard C
机构信息
Department of Dermatology, The University of Texas Southwestern Medical Center, Dallas, Texas, USA; Medical Scientist Training Program, The University of Texas Southwestern Medical Center, Dallas, Texas, USA; Harmon Center for Regenerative Science and Medicine, The University of Texas Southwestern Medical Center, Dallas, Texas, USA.
Department of Biomedical Engineering, The University of Texas Southwestern Medical Center, Dallas, Texas, USA; Department of Biochemistry, The University of Texas Southwestern Medical Center, Dallas, Texas, USA; Program in Genetic Drug Engineering, The University of Texas Southwestern Medical Center, Dallas, Texas, USA; Harold C. Simmons Comprehensive Cancer Center, The University of Texas Southwestern Medical Center, Dallas, Texas, USA.
出版信息
J Invest Dermatol. 2025 Apr;145(4):780-789. doi: 10.1016/j.jid.2024.07.029. Epub 2024 Sep 14.
Advances in sequencing technologies have facilitated the identification of the genes and mechanisms for many inherited skin diseases. Although targeted nucleic acid therapeutics for diseases in other organs have begun to be deployed in patients, the goal of precise therapeutics for skin diseases has not yet been realized. First, we review the current and emerging nucleic acid-based gene-editing and delivery modalities. Next, current and emerging viral and nanoparticle vehicles for the delivery of gene therapies are reviewed. Finally, specific skin diseases that could benefit optimally from nucleic acid therapies are highlighted. By adopting the latest technologies and addressing specific barriers related to skin biology, nucleic acid therapeutics have the potential to revolutionize treatments for patients with skin disease.
测序技术的进步推动了许多遗传性皮肤病相关基因和发病机制的鉴定。尽管针对其他器官疾病的靶向核酸疗法已开始应用于患者，但皮肤病精准治疗的目标尚未实现。首先，我们回顾当前和新兴的基于核酸的基因编辑和递送方式。接下来，对当前和新兴的用于基因治疗递送的病毒和纳米颗粒载体进行综述。最后，重点介绍了最能从核酸疗法中获益的特定皮肤病。通过采用最新技术并解决与皮肤生物学相关的特定障碍，核酸疗法有可能彻底改变皮肤病患者的治疗方式。
Zotero 插件