Modulation of LMNA splicing as a strategy to treat prelamin A diseases

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Modulation of LMNA splicing as a strategy to treat prelamin A diseases.

The alternatively spliced products of LMNA, lamin C and prelamin A (the precursor to lamin A), are produced in similar amounts in most tissues and have largely redundant functions. This redundancy suggests that diseases, such as Hutchinson-Gilford progeria syndrome (HGPS), that are caused by prelamin A-specific mutations could be treated by shifting the output of LMNA more toward lamin C. Here,...

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ژورنال

عنوان ژورنال: Journal of Clinical Investigation

سال: 2016

ISSN: 0021-9738,1558-8238

DOI: 10.1172/jci85908