Gene therapy

8 papers1 finding

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.

On aging: the drug’s trials ARN judged to be about aging, of every trial that tests it for any use.

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.

Funders: National Institute on Aging and others

Outcomes

Atherogenic lipids

downin humans1

1 study

Gene therapy decreases atherogenic lipids in humans.
“Techniques including CRISPR-Cas9 gene editing, small interfering RNA, and antisense oligonucleotides enable precise modulation of key genes such as PCSK9, ANGPTL3, APOC3, and LPA, leading to sustained reductions in atherogenic lipids.”
Reviewin dyslipidemia
Gene Therapy for Lipid Management: Dawn of a New Era
Cureus11 Jul 2026