Human laminin-111 and laminin-211 protein therapy prevents muscle disease progression in an immunodeficient mouse model of LAMA2-CMD.

ABSTRACT:

Background

Laminin-α2-related congenital muscular dystrophy (LAMA2-CMD) is a devastating genetic disease caused by mutations in the LAMA2 gene. These mutations result in progressive muscle wasting and inflammation leading to delayed milestones, and reduced lifespan in affected patients. There is currently no cure or treatment for LAMA2-CMD. Preclinical studies have demonstrated that mouse laminin-111 can serve as an effective protein replacement therapy in a mouse model of LAMA2-CMD.

Methods

In this study, we generated a novel immunocompromised dy^W mouse model of LAMA2-CMD to study the role the immune system plays in muscle disease progression. We used this immune-deficient dy^W mouse model to test the therapeutic benefits of recombinant human laminin-11

SUBMITTER: Barraza-Flores P 

PROVIDER: S-EPMC7271547 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature