Experimental animal model

Cell Type/delivery

Important findings

Teratogenicity

References

1

Rat model of traumatic spinal injury. Cell transplantation at 9 days after injury.

Mouse ESC derived neural cells/ Direct transplantation at the injured site.

Survival of the transplanted cells until 5 weeks of observation and differentiation into astrocytes, oligodendrocytes and neurons.

Not reported

McDonald et al., 1999.

2

SCID mice

hESCs derived PECAM+ cells. PLGA/PLLA patch or scaffold based s.c implantation.

New micro vessels with human endothelial cells lining observed on day 7 and 14 after engraftment.

Not reported

Levenberg et al., 2002

3

(MPTP-treated) monkeys, a primate model for Parkinson’s disease

Dopaminergic neurons from monkey ESC derived neurospheres treated with FGF20 and FGF2 to for differentiation. Direct injection into bilateral putamen.

Functional engraftment of dopaminergic neurons to alleviate MPTP-induced neurological symptoms

Not reported

Takagi et al., 2005

4

Athymic male rat heart model of I/R injury.

hESC derived cardiomyocytes and treated with pro-survival cocktail. Cell transplantation 4 days after I/R injury by direct intramyocardial injection.

Engraftment and survival and integration of the transplanted cells. Significant attenuation of LV remodeling and preservation of cardiac function

Not reported

Laflamme et al., 2005

5

Mouse model of myogenesis using cardiotoxin injection alone or with 25 Gy X-radiation.

hESCs derived myogenic precursors/IM injection.

Myogenic differentiation of xenotransplanted precursors.

No evidence of teratogenicity until 128 days of observation.

Zheng et al., 2006

6

Rat model of myocardial infarction by coronary artery ligation with immunosuppression.

Direct intra-myocardial injection of hESC derived CMs and Non-CMs.

Integration of hESCs derived CMs with host CMs via gap junctions, attenuated LV-remodeling and improved LVFS.

No teratomas observed in hESC derived CMs.

Caspi et al., 2007

7

Porcine model of retinal stem cell transplantation prepared by treatment of iodoacetic acid to eliminate photoreceptors

Swine iPSCs derived Rho + rod photoreceptors transplanted in to sub-retinal space of pig eye.

Successful engraftment and integration of iPSCs-derived Rho + photoreceptors into damaged neural retina of pig.

Not reported

Zhou et al., 2011

8

SCID/beige mice (deficient in B and T lymphocytes and NK cells) with alloxan induced diabetes. Subcutaneous implantation model.

hESCs and their derivative pancreatic islet progenitor cells loaded into bilaminar theracytes. Subcutaneous implantation of the loaded theracytes,

Cells remain fully encapsulated. The derivative pancreatic islet cells secreted sufficient insulin to alleviate diabetes at 20 weeks after implantation.

Not reported

Kirk et al., 2014

9

Macaque myocardial model of ischemia followed by reperfusion.

hESCs derived CM intramyocardial delivery

Extensive re-muscularization of myocardium and electro- mechanical junctions between donor and host myocytes. Non-fatal arrhythmias.

Not observed until 84 days of observation.

Chong et al., 2014

10

Patients with Stargardt’s macular dystrophy and dry age-related macular degeneration

hESC

retinal pigmented epithelium (RPE)

absent in an observation period of 4 months

Schwartz, 2012