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Large‐scale functional LIPA variant characterization to improve birth prevalence estimates of lysosomal acid lipase deficiency

Lysosomal acid lipase (LAL) deficiency is an autosomal recessive disorder caused by LIPA gene mutations that disrupt LAL activity. We performed in vitro functional testing of 149 LIPA variants to increase the understanding of the variant effects on LAL deficiency and to improve disease prevalence es...

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Bibliografiske detaljer
Udgivet i:Hum Mutat
Main Authors: del Angel, Guillermo, Hutchinson, Andrew T., Jain, Nina K., Forbes, Chris D., Reynders, John
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2019
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6852163/
https://ncbi.nlm.nih.gov/pubmed/31180157
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/humu.23837
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