Progerin mRNA
2 papers2 findings
- Animals2
Interventions
1SRP-2001
downin human cells1downin mice1
1 study
SRP-2001
downin human cells1downin mice1
SRP-2001 decreases progerin mRNA in human cells.
“PPMO SRP-2001 provided the most significant decrease in progerin transcripts in patient fibroblasts”
Cellspatient fibroblasts
A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome
Nature medicine11 Mar 2021
SRP-2001 decreases progerin mRNA in mice.
“Intravenous delivery of SRP-2001 to a transgenic mouse model of HGPS produced significant reduction of progerin transcripts in the aorta”
Animalsintravenous deliveryaorta
A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome
Nature medicine11 Mar 2021
Latest
Molecular Therapy
Precise progerin targeting using RfxCas13d: A therapeutic avenue for Hutchinson-Gilford progeria syndrome
Experiments in miceProgeria
Nature Medicine
A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome
Experiments in miceProgeria
PLOS One
The mutant form of lamin A that causes Hutchinson-Gilford progeria is a biomarker of cellular aging in human skin
Cross-sectional study of 150 peopleBiomarkers