Table 2.

Summary of important advances in mRNA therapeutics for monogenic diseases.

Disease (Gene)	Method	Result	Model
CF (CFTR)	Transfection with optimized wt-CFTR mRNA	Increase in functional CFTR protein	Cells [50]
	LNP-packaged modified CFTR mRNA	Increase in CFTR protein function	Patient-derived cells, mice [51]
	ReCode RTX00001 mRNA in SORT-LNP	Restoration of CFTR function	Primary hBE cells and mice [52]
	Arcturus LUNAR-LNP system	Significant in vivo functional restoration	Ferret and mice in [53]
	Nebulized MRT5005 mRNA	No adverse effects	First-in-human phases 1/2 trial [55,56]
Haemophilia B (hEPO, hFIX)	LNPs and Lipid-like nanoparticle delivery of mRNAs	Production of a “depot” system of functional protein production in hepatocytes	Mice and non-human primates [58]
MMA (hMUT)	Intravenous administration of hMUT LNP-encapsulated mRNAs	Increased liver production of active MUT, survival and weight gain.	Mice [59,60]
Arginase 1 deficiency (ARG1)	Injection of LNP-encapsulated human codon-optimised mRNA	Increased liver hARG1 expression and activity, increased survival.	Mice [62,63]