神经学孤儿疾病:新兴创新和基因治疗的作用

Ivelina P Kioutchoukova1, Devon T Foster2, Rajvi N Thakkar1

  • 1College of Medicine, University of Florida, Gainesville, FL 32611, United States.

概括

基因疗法,包括CRISPR-Cas9和病毒载体,对治疗影响肌肉,认知和神经元的罕见神经系统孤儿疾病充满希望. 这些创新的基因治疗方法为患者提供了新的希望.

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