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The findings, published on February 18, 2019 in the journal Nature Medicine, highlight a novel CRISPR/Cas9 genome-editing therapy that can suppress the accelerated aging observed in mice with Hutchinson-Gilford progeria syndrome, a rare genetic disorder that also afflicts humans. This treatment provides important insight into the molecular pathways involved in accelerated aging, as well as how to reduce toxic proteins via gene therapy.
"Aging is a complex process in which cells start to lose their functionality, so it is critical for us to find effective ways to study the molecular drivers of aging," says Juan Carlos Izpisua Belmonte, a professor in Salk's Gene Expression Laboratory and senior author of the paper. "Progeria is an ideal aging model because it allows us to devise an intervention, refine it and test it again quickly."
Onset - Progression - Progeria - Forms - Group
With an early onset and fast progression, progeria is one of the most severe forms of a group of degenerative disorders caused by a mutation in the LMNA gene. Both mice and humans with progeria show many signs of aging, including DNA damage, cardiac dysfunction and dramatically shortened life span. The LMNA gene normally produces two similar proteins inside a cell: lamin A and lamin C. Progeria shifts the production of lamin A to progerin. Progerin is a shortened, toxic form of lamin A that accumulates with age and is exacerbated in those with progeria.
"Our goal was to diminish the toxicity from the mutation of the LMNA gene that leads to accumulation of progerin inside the cell," says co-first author Hsin-Kai Liao, a staff researcher in the Izpisua Belmonte lab. "We reasoned that progeria could be treated by CRISPR/Cas9-targeted disruption of both lamin A and progerin."
Researchers - CRISPR/Cas9 - System - Gene - Therapy
The researchers utilized the CRISPR/Cas9 system to deliver the gene therapy into the cells of the progeria mouse model expressing Cas9. An adeno-associated virus (AAV)...
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