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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5 factors to consider when planning a stem cell reprogramming project

When you are planning a project involving stem cell reprogramming, there are several factors to consider when choosing a reprogramming technology and a service provider. This post will highlight 5 questions you may want to answer before choosing a service provider or reprogramming technology.

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How to determine CRISPR gene editing success in 2 weeks

Imagine CRISPR – but more efficient, reliable and flexible. SNIPER works in synergy with the CRISPR-Cas9 system to fulfill even the most challenging gene editing projects – including biallelic modifications, single base-pair changes and large DNA insertions. Read on to discover four examples of CRISPR-SNIPER in action.

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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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Stem Cells for Autism

In this post, we highlight recent advancements in stem cells as treatment and study of autism to commemorate World Autism Awareness Day (2 April 2019).

Autism spectrum disorder (ASD) is a complex developmental disability that impairs the ability to communicate and relate to others. Affecting 1% of the world population, it is the most commonly diagnosed childhood developmental disorder. In 2018, Centers for Disease Control (CDC) reported that rates of autism had increased to an estimated 1 in 59 children, up from 1 in 88 just six years ago.

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Spotlight: StemRNA Neuro iPSCs – Human Brain Neurons for Functional Assays

Human induced pluripotent stem cell (iPSCs) derived  neural models to study brain disease provide an unlimited resource for disease modelling as well as being a tool for drug screening for effective therapies. The limited access to viable patient neurons from brain tissue itself generates the need for functional and reproducible human neuron cell models. Such cells are now increasingly used for drug development studies as well as supporting research into mechanisms and pathways of various neurological diseases.

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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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Tissue Sourcing for the Development of iPSC-derived Disease Models

Human induced pluripotent stem cells (iPSCs)-based models are an ideal resource for studying disease mechanisms in vitro at the cellular level[1], screening potential new therapeutics[2], and investigating the propensity and mechanism for the development of toxic side effects caused by drug treatment[3]. Such iPSC-based models enable studies to be performed under defined experimental conditions and in a reproducible manner.

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