Crispr/cas9-mediated disruption of the cryptic splice site

1 paper3 findings

C, W, M, H: cells, worms or flies, mice, humans. Dark: independent labs found the same change there. Light: one lab or one study. Outline: nothing yet.

Labs are independent when their papers share no last author and no institution.

Against: findings that point the other way from most findings on the same outcome.

No results posted: trials that ended more than a year ago with no results on ClinicalTrials.gov and no paper in ARN’s record.

Company ties: findings with an author at a company, or from a paper whose competing-interests statement declares ties to one.

1 lab in 2 countries

Animals1

Outcomes

Multiorgan pathology

downin mice1

1 study

Crispr/cas9-mediated disruption of the cryptic splice site decreases multiorgan pathology in mice.
Animalsin Cas9-expressing LmnaG609G/G609G mice
Genomic splice-donor disruption rescues systemic disease in progeria
bioRxiv18 Sep 2026Preprint

Progerin

downin human cells1

1 study

Crispr/cas9-mediated disruption of the cryptic splice site decreases progerin in human cells (by 92.7-96.8%).
Cellspatient-derived fibroblasts and iPSC-derived MSCs
Genomic splice-donor disruption rescues systemic disease in progeria
bioRxiv18 Sep 2026Preprint

Latest

1 in 12 months

bioRxiv18 Sep

Genomic splice-donor disruption rescues systemic disease in progeria

Preprint: experiments in miceProgeria

Paper

Crispr/cas9-mediated disruption of the cryptic splice site

Outcomes

Latest

Genomic splice-donor disruption rescues systemic disease in progeria

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