Table 7.

A summary of the elements used in developing non-integrating lentiviral vectors (NILVs) for site-directed integration in pre-clinical studies. Different approaches for direct integration or site-specific modifications of safe genomic loci in different studies are listed with attached references.

NILV Modification	Disease/Application	Transgene/Effector	Target	References
D64V	β-thalassemia/site-directed integration and KLF1 gene modification	Zinc finger nuclease/ZFN donor template–GFP expression cassette	γ-globin and HbF expression	[112]
D64V	β-thalassemia/site-specific gene modification	Zinc finger nuclease/ZFN donor template	SOX6 region	[113]
D64V	Fanconi anemia/gene targeting using ZFN and IDLVs	OCT4, SOX2, c-MYC, and KLF4 flanked by loxP sequences.	AAVS1 safe harbor locus in fibroblasts	[114]
D64V	Site-specific gene modification	Transcription activator-like effector nucleases/TALEN donor template	COL7A1 gene	[115]