Table 1.

Genome editing studies for Mucopolysaccharidoses.

Pre-Clinical Studies in Cell Models
Disease	Affected Gene	Targeted Gene	Platform	Cell Type		Delivery Method			Genetic Modification	Reference
MPS I	IDUA	IDUA	CRISPR/Cas9	Human fibroblasts		Plasmid-Liposome complex			SNV correction	[82]
MPS I	IDUA	IDUA	CRISPR/Cas9	Human fibroblasts		Plasmid-Liposome complex			SNV correction	[93]
MPS I	IDUA	IDUA	CRISPR/Cas9	mouse iPSCs		Plasmid-Liposome complex			Precise deletion	[92]
Pre-Clinical Studies in Murine Models
Disease	Affected Gene	Targeted Gene	Platform	In Vivo Vs. Ex Vivo	Cargo and Vehicle			Genetic Modification		Reference
MPS I	IDUA	CCR5	CRISPR/Cas9	ex vivo	RNP/AAV6			Gene addition		[90]
MPS I	IDUA	ROSA26	CRISPR/Cas9	in vivo	Liposome and plasmid vectors, IV			Gene addition		[84]
MPS I	IDUA	IDUA	CRISPR/Cas9	in vivo	2 AAV9 vectors, IV			SNV correction		[86]
MPS I	IDUA	ALB	ZFNs	in vivo	3 AAV2/8 vectors, IV			Gene addition		[78]
MPS II	IDS	ALB	ZFNs	in vivo	3 AAV2/8 vectors, IV			Gene addition		[77]
Clinical Trials
Disease	Affected Gene	Targeted Gene	Platform	In Vivo vs. Ex Vivo	Cargo and Vehicle	Genetic Modification	Company		Trial Name	Clinicaltrials.gov Identifier
MPS I	IDUA	CCR5	CRISPR/Cas9	ex vivo	RNP/AAV6	Gene addition	Stanford University		in the pipeline
MPS I	IDUA	ALB	ZFNs	in vivo	3 AAV2/6 vectors, IV	Gene addition	Sangamo therapeutics		SB-318	NCT02702115
MPS II	IDS	ALB	ZFNs	in vivo	3 AAV2/6 vectors, IV	Gene addition	Sangamo therapeutics		SB-913	NCT3041324
IV: intravenous