Cell mediated gene therapy: A guide for doctors in the clinic

Table 1 Current cell mediated gene therapy protocols in clinical and R and D stages

Target disease	Stage	Gene modification	Target cell	Ref.
Hemophilia A	Preclinical	Oct4, Sox2, Klf4	Fibroblast	Xu et al[40]
ALS	Preclinical	Oct4, Sox2, Klf4, c-Myc	Fibroblast	Dimos et al[55]
Alzheimer	Preclinical	Oct4, Sox2, Klf4, c-Myc	Fibroblast	Israel et al[46]
ALS	Preclinical	Oct4, Sox2, Klf4	Fibroblast	Chen et al[54]
Cardiac failure	Preclinical	Gata4, Mef2c, Tbx4	Fibroblast	Ieda et al[22]
Diabetes	Preclinical	Oct4, Sox2, Klf4	Fibroblast	Zhang et al[24]
Schizophrenia	Preclinical	Oct4, Sox2, Klf4, c-Myc	Fibroblast with 15q11.2 del	Yoon et al[12]
Barth syndrome	Preclinical	Oct4, Sox2, Klf4, c-Myc	Fibroblast	Wang et al[52]
Parkinson’s disease	Preclinical	Oct4, Sox2, Klf4, c-Myc	Fibroblast	Chung et al[50]
Macular degeneration	PhaseI	Oct4, Sox2, Klf4, c-Myc	Fibroblast	Kamao et al[14]
Degenerative arthritis	Phase II/III	TGF-β1	Chondrocyte	Ha et al[15]
X-linked adrenoleukodystrophy	In the clinic	ABCD1	CD34+ Bone marrow cell	Cartier et al[36]
Wiskott-aldrich syndrome	In the clinic	WASP	CD34+ Bone marrow cell	Aiuti et al[35]
Metachromatic leukodystrophy	In the clinic	ARSA	CD34+ Bone marrow cell	Biffi et al[37]

ALS: Amyotrophic lateral sclerosis; TGF-β: Transforming growth factor-beta.