Hutchinson-Gilford Progeria paves the way for novel targeted anti-aging therapies

Oliver Dreesen; Brian Kennedy

doi:10.1016/j.medj.2021.03.005

Hutchinson-Gilford Progeria paves the way for novel targeted anti-aging therapies

Med. 2021 Apr 9;2(4):353-354. doi: 10.1016/j.medj.2021.03.005. Epub 2021 Apr 9.

Authors

Oliver Dreesen¹, Brian Kennedy²

Affiliations

¹ Cell Aging, Skin Research Institute of Singapore, A∗STAR, 8A Biomedical Grove, #06-06 Immunos, Singapore, 138648, Singapore. Electronic address: [email protected].
² Healthy Longevity Translational Research Programme, Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore; Departments of Biochemistry and Physiology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore, Singapore; Centre for Healthy Longevity, National University Health System, Singapore, Singapore; Singapore Institute of Clinical Sciences, A∗STAR, Singapore, 117609, Singapore.

PMID: 35590156
DOI: 10.1016/j.medj.2021.03.005

Abstract

Hutchinson-Gilford Progeria is an accelerated aging syndrome caused by permanently farnesylated mutant lamin A, termed progerin. Recently, the FDA approved Lonafarnib, a farnesyltransferase inhibitor, to treat progeria, while Koblan and colleagues used novel gene editing methods to target the root cause of this disease by correcting the LMNA mutation.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Aging
Enzyme Inhibitors / therapeutic use
Humans
Lamin Type A / genetics
Mutation
Progeria* / drug therapy

Substances

Enzyme Inhibitors
Lamin Type A