Researchers at the Experimental and Clinical Research Center in Berlin are developing a targeted treatment for muscular dystrophy with the help of gene ... To fix the dysferlin mutation, Escobar uses ...

For nearly 20 years, Spuler and her collaborators have been working to understand dysferlin, its role in muscular dystrophy and ways to cure these ... Escobar uses CRISPR-Cas9, which is often ...

Several companies are using CRISPR to edit human genomes in an attempt to treat (and even cure) genetic diseases ... including cystic fibrosis and Duchenne muscular dystrophy.

If you buy through a BGR link, we may earn an affiliate commission, helping support our expert product labs. Researchers in New Zealand have kicked off a new trial for a CRISPR-based cure for high ...

A patient would only need a single treatment. CRISPR-Cas9 is comprised of ... is pursuing treatments for cystic fibrosis. Duchenne Muscular Dystrophy (DMD), hemophilia A and B, HIV, inherited ...

Understanding Rare Diseases: We will begin by shedding light on the nature of rare diseases, their rarity, and the immense challenges they pose to diagnosis and treatment. 2. The Promise of CRISPR: We ...