Table 3. [Potential Treatments Tested Only In Vitro and/or in Murine Studies]. - GeneReviews®

Treatment/Drug	Pathway	Target
All-trans retinoic acid	Autophagy	Progerin turnover
Antisense oligonucleotides	Access of splicing machinery	Lamin C / prelamin A splicing &/or abnormal LMNA splicing
DOT1L inhibitors	Cell reprogramming	DOT1L
Isoprenylcysteine carboxyl methyltransferase (ICMT) knock-down = shICMT	Prelamin A processing	ICMT
Gene editing	CRISPR/Cas9	LMNA sequence
JH4 & progerinin	Progerin-lamin A/C binding	Progerin-lamin A/C binding
Metformin	Activation of AMPK	Hepatic gluco-neogenesis
Methylene blue	Mitochondrial biogenesis	Mitochondria function
MG132 (a proteasome inhibitor )	Autophagy	Progerin turnover
Mono-aminopyrimidines	Prelamin A processing	Prelamin A farnesylation
N-acetyl cystine	Oxidative stress	Reactive oxygen species
caNRF2	NRF2 reactivation	NRF2
OSKM induction	Epigenetic remodeling	Partial cellular reprogramming
Pyrophosphate	Metabolism of extracellular pyrophosphate	Calcium-phosphate deposition
Rapamycin & rapalogs	Autophagy	Progerin turnover
Remodelin	Microtubule	NAT10
Resveratrol	SIRT1 activity	SIRT1
Sodium salicylate	NF-κB signaling	NF-κB inhibition
Stem cell transplantation	Stem cell function	Tissue regeneration
Sulforaphane	Autophagy	Progerin turnover
Telomerase	Telomere length	Telomeres ¹
Vitamin D	Vitamin D receptor signaling	Vitamin D receptor

Treatment/Drug

Pathway

Target

All-trans retinoic acid

Autophagy

Progerin turnover

Antisense oligonucleotides

Access of splicing machinery

Lamin C / prelamin A splicing &/or abnormal LMNA splicing

DOT1L inhibitors

Cell reprogramming

DOT1L

Isoprenylcysteine carboxyl methyltransferase (ICMT) knock-down = shICMT

Prelamin A processing

ICMT

Gene editing

CRISPR/Cas9

LMNA sequence

JH4 & progerinin

Progerin-lamin A/C binding

Metformin

Activation of AMPK

Hepatic gluco-neogenesis

Methylene blue

Mitochondrial biogenesis

Mitochondria function

MG132 (a proteasome inhibitor )

Autophagy

Progerin turnover

Mono-aminopyrimidines

Prelamin A processing

Prelamin A farnesylation

N-acetyl cystine

Oxidative stress

Reactive oxygen species

caNRF2

NRF2 reactivation

NRF2

OSKM induction

Epigenetic remodeling

Partial cellular reprogramming

Pyrophosphate

Metabolism of extracellular pyrophosphate

Calcium-phosphate deposition

Rapamycin & rapalogs

Autophagy

Progerin turnover

Remodelin

Microtubule

NAT10

Resveratrol

SIRT1 activity

SIRT1

Sodium salicylate

NF-κB signaling

NF-κB inhibition

Stem cell transplantation

Stem cell function

Tissue regeneration

Sulforaphane

Autophagy

Progerin turnover

Telomerase

Telomere length

Telomeres ¹

Vitamin D

Vitamin D receptor signaling

Vitamin D receptor

From: Hutchinson-Gilford Progeria Syndrome

GeneReviews^® [Internet].

Adam MP, Feldman J, Mirzaa GM, et al., editors.

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