Systemic AAV-Delivered Targeted Gene Editing Restores Cerebrovascular Disease and Prevents Associated Neurodegeneration in ACTA2 R179H-Related Multisystemic Smooth Muscle Dysfunction Syndrome (MSMDS)

Vijai Krishnan, Sabyasachi Das, Christiano Alves,Christian Lino Cardenas,Takahiko Imai, James Hai, Aarushi Gandhi, Pazhanichamy Kalailingam, David Chung, Mark E. Lindsay,Benjamin P. Kleinstiver,Patricia L. Musolino

MOLECULAR THERAPY（2024）

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