Rare diseases affect 30 million people in the USA and more than 300–400 million
worldwide, often causing chronic illness, disability, and premature death. Traditional …

The synergism of stem cell biology and biomaterial technology promises to have a profound
impact on stem-cell-based clinical applications for tissue regeneration. Biomaterials …

Friedreich ataxia (FRDA) is an autosomal-recessive disorder characterized by progressive
neurological and cardiac abnormalities. It predominantly affects individuals of European and …

Friedreich's ataxia (FRDA) is an autosomal-recessive neurodegenerative and cardiac
disorder which occurs when transcription of the FXN gene is silenced due to an excessive …

With the discovery of induced pluripotent stem (iPS) cells, it is now possible to convert
differentiated somatic cells into multipotent stem cells that have the capacity to generate all …

Background Human-induced pluripotent stem cell (h-iPSC)–derived cardiac myocytes are a
unique model in which human myocyte function and dysfunction are studied, especially …

Friedreich's ataxia (FRDA) is a recessive neurodegenerative disorder commonly associated
with hypertrophic cardiomyopathy. FRDA is due to expanded GAA repeats within the first …

Among the disciplines of medicine, the study of neurological disorders is particularly
challenging. The fundamental inaccessibility of the human neural types affected by disease …

Objective: Friedreich ataxia (FA) is the most common ataxia and results from an expanded
GAA repeat in the first intron of FXN. This leads to epigenetic modifications and reduced …

With 100 billion neurons and 100 trillion synapses, the human brain is not just the most
complex organ in the human body, but has also been described as “the most complex thing …