靶点背景Lamins are a class of intermediate filament proteins that form a matrix on the inner surface of the nuclear envelope. These proteins are found in many different cell types in three different forms (A, B, and C). Lamins A and C are alternatively spliced versions of the LMNA gene. The LMNA gene has been linked to many disorders of the muscular system, nervous system, and the fat distributions systems including: Emery-Dreifuss muscular dystrophy, Dunnigan-type familial partial lipodystrophy (FPLD), limb-girdle muscular dystrophy (LGMD1B), dilated cardiomyopathy (CMD1A), axonal neuropathy (Charcot-Marie-Tooth disease; CMT2B1), and mandibuloacral dysplasia (MAD).
