Table 3. PB transposon application in gene therapy research.

Target cells/organs	Diseases	Species of cell origin	Therapeutic genes	Gene transfer approach	Improvements			Remarks	References
					Transposon	Transposase	Others
Clinically relevant cells
(i) Induced pluripotent stem cell	α1-antitrypsin deficiency	Human	α-1 antitrypsin	Electroporation		hyPBase	Use with ZFN		[203]
	β-thalassemia	Human	β-globin	Electroporation		hyPBase	Use with CRISPR/Cas9		[147]
	SCD	Human	β-globin	Electroporation		hyPBase	Use with TALEN		[146]
(ii) Mesoangioblast	Duchenne muscular dystrophy	Dog	Microdystrophin	Electroporation		hyPBase			[30]
(iii) T lymphocyte	Hematological malignancies, HER2-specific cancer	Human	Chimeric antigen receptor	Electroporation		hyPBase			[130,131,204–206]
Organs
(i) Kidney	Unilateral ureteral obstruction	Mouse	Insulin-like growth factor-1 receptor	HD					[207,208]
			Glutathione transferase isozyme A4
(ii) Liver	Hemophilia A	Mouse	Factor VIII	HD		hyPBase			[70,122]
	Hemophilia B	Mouse	Factor IX	HD	IRmut	mPBase			[43,121]
				Ultrasound + microbubble	IRmicro	hyPBase
	von Willebrand disease	Mouse	von Willebrand factor	HD		hyPBase			[209]
(iii) Solid tumor	Cervical and ovarian cancer	Mouse	HSV thymidine kinase	Local infusion + PEI					[210,211]

*If not specifically indicated, two-component PB transposon system was used in conjunction with wild-type PBase. Abbreviations: CRISPR/Cas9, clustered regularly interspaced short palindromic repeats/CRISPR-associated protein-9 nuclease; HSV, herpes simplex virus; MAB, mesoangioblast; SCD, sickle cell disease; TALEN, transcription activator like effector nuclease; ZFN, zinc finger nuclease.