Parthenogenesis could treat Inherited Disease, International Stem Cell (ISCO.OB)
The use of parthenogenetic stem cells as a potential strategy to treat genetic diseases such as Huntington’s disease, beta thalassemia or tuberous sclerosis has been validated by researchers at Nationwide Children’s Hospital.
- ISCO.OB created and patented the first proven technology to create human parthenogenetic stem cell lines from unfertilized human eggs;
- ISCO’s technology has similar potential advantages as those described in the work done at Nationwide Children’s Hospital; the creation of human parthenogenetic stem cells that could eliminate a defective copy of a gene and can be immune-matched to the egg donor.
In a scientific paper entitled, “Gene therapy by allele selection in a mouse model of beta-thalassemia” recently published in the Journal of Clinical Investigation describe how they used a mouse model of an inherited blood disorder to show that parthenogenesis could be used to create “genetically clean” stem cells.
These parthenogenetic stem cells demonstrated the ability to correct certain symptoms of such a disease.
- ISCO is particularly pleased to note this animal model research into the potential of parthenogenetic stem cells since ISCO has created method for creating immortal human parthenogenetic stem cell (hpSC) lines;
- These achievements open up the opportunity to assess the efficacy and safety of using parthenogenetic stem cells as a source to create neural cells for the treatment of diseases such as Parkinson’s, Alzheimer’s, and other neurodegenerative disorders.
ISCO expects that the transition from animal models to human therapy using parthenogenetic cells will continue to evolve.
- ISCO has initiated a number of pre-clinical animal studies utilizing both hepatocytes and separately retina pigment epithelium cells, derived from hpSC;
- ISCO’s technology has the additional ethical advantage of not harming a viable human embryo.
The team of researchers were from the Center for Molecular and Human Genetics (Nationwide Children’s Hospital, Columbus, OH), the University of Pennsylvania, the University of North Carolina and the University of Minnesota led by John McLaughlin Ph.D.
