Congenital, Hereditary, and Neonatal Diseases and Abnormalities: News and Research on Muscular Dystrophies, Limb-Girdle

MeSH tree: C16.320.577.280

CRISPR therapy corrects muscular dystrophy mutations and regrows muscles in mice

Researchers at the Experimental and Clinical Research Center (ECRC), a joint institution of the Max Delbrück Center and Charité—Universitätsmedizin Berlin, have developed a promising gene-editing approach intended to restore ...

Jan 7, 2025

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Next-gen gene therapy vector for muscle diseases uses AI predictive methodology to improve efficacy and safety

A new study describes the use of artificial intelligence (AI) in designing a new generation of capsids, the structures that envelop genetic material of adeno-associated viruses (AAV), to improve gene therapies for muscle ...

Sep 12, 2024

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Genetic discovery leads to diagnoses of 9 people with rare form of muscular dystrophy

Nine people around the world who have been living with unexplained, muscular dystrophy-like symptoms may now have an answer for what is causing the progressive muscle weakness in their body's mid-section, arms and legs.

Jul 17, 2023

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Diagnosis of rare, genetic muscle disease improved by new approach

It's not easy to distinguish between the dozens of subtypes of limb girdle muscular dystrophy—a rare, genetic muscle disease characterized by weakness in the hips and shoulders that causes difficulty walking and lifting the ...

Jun 15, 2023

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