SRP-2001
1 paper4 findings
- 1 lab in 1 country
- Funders: National Heart, Lung, and Blood Institute and 2 more
- 4 of 4 findings with company ties
- Animals1
Outcomes
3Progerin mRNA
downin human cells1downin mice1
1 study
Progerin mRNA
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
Vascular smooth muscle cells
downin mice1
1 study
Vascular smooth muscle cells
downin mice1
SRP-2001 prevents vascular smooth muscle cells in mice.
“rescue of vascular smooth muscle cell loss in large arteries”
Animalsin transgenic mouse model of HGPSlarge arterieslong-term continuous treatment
A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome
Nature medicine11 Mar 2021
Lifespan
upin mice1+62% lifespan
1 study
Lifespan
upin mice1+62% lifespan
SRP-2001 extends lifespan in mice (+61.6% lifespan).
“Long-term continuous treatment with SRP-2001 yielded a 61.6% increase in lifespan”
Animalsin transgenic mouse model of HGPSlong-term continuous treatment
A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome
Nature medicine11 Mar 2021