Question

Give 1 example of induced pluripotent stem cell therapies that are currently under research for medical use (cite scientific studies for this). What steps remain to be accomplished before FDA approval can be given?

Answer 1

Question:

Give 1 example of induced pluripotent stem cell therapies that are currently under research for medical use (cite scientific studies for this). What steps remain to be accomplished before FDA approval can be given?

Answer:

Induced pluripotent stem cell (iPSC) technology, is a method of inducing pluripotency in mature somatic cells by treatment with defined factors (tissue-specific transcription factors), which is now extensively being used in regenerative medicine. Yamanaka and Takahashi in 2006 were the first to generate iPSCs from adult somatic cells by artificially inducing the expression of transcription factors such as OCT4, SOX2, c-MYC, and KLF4 (Yamanaka factors) for which Yamanaka was awarded the Nobel Prize in 2012.

Thalassemia is a genetic blood disorder where mutation in haemoglobin-beta (HBB) gene reduces the synthesis of haemoglobin resulting in decreased red blood cells. This reduction of both haemoglobin and RBCs can be life-threatening.

One of the best approaches in curing thalassemia is to correct the mutation that causes the disease. The patient’s somatic cells can be induced in generating iPSCs which could be used for correcting thalassemia mutation. The promising technology that could be used is the CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats) technology combined with piggyBac transposon, which can efficiently correct HBB mutations in iPSCs with no obvious off-targets. The mutation corrected iPSCs can be differentiated into hematopoietic stem/progenitor cells for transplantation which avoids the problem of transplant rejection.

Most research is done in-vitro which should be translated invivo. Another major hurdle is the cell delivery mode and appropriate application approaches should be established. Before the FDA approval, it has to undergo Phase I, II and III clinical trials before it can be used for medical purposes.