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Myonexus Therapeutics' pipeline includes three clinical stage gene therapy programs (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programs (LGMD2C and LGMD2L).
Finally, a manual filtering step was carried out to prioritize relevant genes in the 30 major LGMD genes for LGMD1B, LGMD2CF, and LGMD2B.
In LGMD2B, a mutation within the DYSF gene abrogates protein expression and is believed to cause the muscle dystrophy .
Since western blot analysis of muscle showed a deficit of dysferlin, the patient was finally diagnosed with LGMD2B.
Several LGMD subtypes have recently been identified based upon mutations of certain genes [7-12]; for example, mutations in the calpain 3 and dysferlin genes cause LGMD2A and LGMD2B, respectively.
Forty-five patients presented with LGMD2B, 31 with MM, 7 with PDM, and 6 were clinically asymptomatic and diagnosed with hyperCKemia.
Dysferlin mutations in LGMD2B, Miyoshi myopathy, and atypical dysferlinopathies.
"CDI's ability to make iPSCs at high quality and purity from any individual, including LGMD2B patients, will provide scientists new tools to understand and address these debilitating forms of muscular dystrophy.
Myonexus' pipeline also includes MYO-102, a gene therapy candidate for LGMD2D (alpha-sarcoglycanopathy) currently completing a Phase 1/2a clinical trial, MYO-201, a gene therapy candidate for LGMD2B (dysferlinopathy) currently in Phase 1, MYO-103, a preclinical gene therapy candidate for LGMD2C (gamma-sarcoglycanopathy), and MYO-301, a preclinical gene therapy candidate for LGMD2L (anoctamin 5).
The company's pipeline includes three clinical stage gene therapy programmes (LGMD2E, LGMD2D, and LGMD2B) and two preclinical gene therapy programmes (LGMD2C and LGMD2LO).
LGMD2B is a recessive genetic disease caused by a toxic loss of function in the dysferlin gene.
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