Abstract 4359043: NVC-001 – An AAV Gene Therapy Functional Cure for LMNA Dilated Cardiomyopathy

Rong Ng, Sheng; Wang, Audrey; Tukijan, Farhana; Aruna Murugiah, Astley; Thing Tan, Liang; Teo, Angela; Yi Lee, Qian; Tan, Hayden; Choy, Audrey; Ong, Mervyn; Hsu, Wan-En; Varsano, Giulia; Pomp, Oz; K. Lee, John; Chong Tan, Yann

doi:10.1161/circ.152.suppl_3.4359043

Public

Abstract 4359043: NVC-001 – An AAV Gene Therapy Functional Cure for LMNA Dilated Cardiomyopathy

Published in Circulation • Nov 3, 2025

Authors:

Sheng Rong Ng

Audrey Wang

Farhana Tukijan

Abstract

Background: LMNA-related Dilated Cardiomyopathy (LMNA-related DCM) is an aggressive type of DCM caused by pathogenic mutations in the LMNA gene that comprises 5-6% of genetic DCM cases. NVC-001 is an adeno-associated virus (serotype 9) gene-therapy vector encoding a truncated form of the naturally o...

Subject

LMNA

Dilated cardiomyopathy

Medicine

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