A humanized knock-in Col6a1 mouse recapitulates a deep-intronic splice-activating variant

Authors


Lab

Journal
bioRxiv

Abstract
Antisense therapeutics such as splice-modulating antisense oligonucleotides (ASOs) are promising tools to treat diseases caused by splice-altering intronic variants. However, their

Keywords/Topics
Humanized mouse model ; Collagen VI - related muscular dystrophies ;COL6A1 ; pseudoexon Introduction Collagen VI - related

BIOSEB Instruments Used:
Grip strength test (BIO-GS4)

Source :

https://pmc.ncbi.nlm.nih.gov/articles/PMC10996637/

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