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dystrophin

British  
/ ˈdɪstrəfɪn /

noun

  1. a protein, the absence of which is believed to cause muscular dystrophy

"Collins English Dictionary — Complete & Unabridged" 2012 Digital Edition © William Collins Sons & Co. Ltd. 1979, 1986 © HarperCollins Publishers 1998, 2000, 2003, 2005, 2006, 2007, 2009, 2012

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The effects were that muscular dystrophin was alleviated throughout the body.

From Science Daily ● Mar. 6, 2024

But because the gene for the missing dystrophin protein is so large, a smaller version of the gene is used.

From Seattle Times ● Jun. 22, 2023

The DNA encoding dystrophin is too large to package into the AAVs widely used in gene therapy.

From Science Magazine ● May 23, 2023

His condition stems from a mutation in a gene that provides instructions for making dystrophin, a protein primarily in muscles used for movement and cardiac muscle.

From Washington Post ● Feb. 5, 2022

Known as exon skipping, this resulted in the downstream mRNA no longer being unreadable and in functional dystrophin being formed.

From Nature ● Oct. 15, 2019

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