Vascular #GeneEditing therapy #MultisystemiSmoothMuscleDysfunctionSyndrome

ABE8e-eVRQR
AAV-PR
MYH11-Cre:ACTA2 R179H/+🐭
P3 iv▶️
8-wk editing
Liver 45%🫀12%🧠vessel 7% Aorta 1%

Lifespan 6➡️23 wk
⬇️Aorticφ
⬆️Locomotor
⬆️🧠Arteriolar SMC

@markelindsay.bsky.social bioRxiv 2024
www.biorxiv.org/content/10.1...