Cell type-specific gene therapy confers protection against motor neuron disease caused by a TFG variant.
细胞类型特异性基因疗法可预防由 TFG 变异引起的运动神经元疾病
期刊:Proceedings of the National Academy of Sciences of the United States of America
影响因子:9.1
doi:10.1073/pnas.2410996121
Lettman Molly M, Mendina Caitlin A, Burkard Emma, Alvin James R, Zhu Yunyun, Coon Joshua J, Audhya Anjon
