Stem cells offer hope for sufferers of Spinocerebellar Ataxia

What is Spinocerebellar Ataxia?

Spinocerebellar ataxias (SCAs) are a group of inherited, genetically heterogeneous disorders which are characterised by ocular motor abnormalities, cognitive dysfunction, peripheral neuropathy and progressive cerebellar ataxia. In general, the prevalence of SCA is between 2-7/100,000 individuals with more than 30 subtypes being identified, each caused by a mutation of a different gene.

PolyQ SCAs including SCA1, SCA2, SCA3, SCA6, SCA7 and SCA17 are caused by an extensive CAG sequence repeat which encodes for expanded polyglutamine residues within the ATXN3 gene. The only currently approved treatment option for patients suffering with this debilitating, progressive disease is Ceredist, however this only provides palliative treatment in reducing or relieving frequency and severity of symptoms. Stem cell therapy is offering hope, with evidence of potential efficacy being demonstrated in a phase I/II clinical trial using stem cells isolated and cultured from human adipose tissue.

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How to mass culture iPSCs in suspension

Induced pluripotent stem cells (iPSCs) have the potential to revolutionize the fields of drug discovery, disease modelling, personalized medicine and regenerative medicine [1]. iPSCs have the capacity for continuous self-renewal and differentiation into several different cell types. At the same time, they also do not face many of the ethical challenges faced by human embryonic stem cells [2].

In order to fully realize the potential of iPSCs for many of the above applications, there is a requirement to generate a vast number of cells; hence, an effective method of mass culture of iPSCs is highly sought after by iPSC researchers.

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Pluripotent Potential for Clinical Application

Since their development in the mid-2000s, the versatile nature of induced Pluripotent Stem Cells (iPSCs) has unlocked the potential of curative approaches instead of symptom-reactive treatments. In particular, fields which deal with genetic disorders and regenerative therapies would benefit from this. The advancement of iPSC technology compliments the advent of personalized medicine, allowing for a future where individuals could be treated using autologous iPSCs.

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Using human iPSCs as an in vitro model for regenerative medicine, drug screening and disease modelling

In translational medicine, research using primary patient samples is advantageous over cell lines as the expression of the protein under investigation is regulated by native elements in primary samples.

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