Myotonic dystrophies (DMs) are pleotropic multisystemic diseases. These dominantly transmitted repeat disorders affect multiple organs of the human body at all ages—from the newborns to the elderly. DMs are highly inconsistent in terms of age at onset, severity of symptoms, and clinical patterns. Even within families, the onset and pattern of organ involvement remains enigmatic. Anticipat…
While most stem cell books focus on basic aspects and/or cell therapy, this book emphasizes the relevance of stem cells obtained from patients, the so-called “patients in a petri dish” as tools to investigate human genetic diseases for which there are no available effective treatment. Chapters embrace several examples of the use of iPS cell technology, a recent Nobel Prize-winning scientifi…