Textbook of Personalized Medicine - Second Edition [2015]

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Personalized Cell Therapy for PD

Various types of cell therapy have been used for PD but none of these has so far
proven entirely satisfactory. Rejection of transplanted cells is one problem. Use of
iPSCs for personalized cell therapy of PD is feasible without problem of tissue
rejection (Fig. 12.3 ).

Personalized Management of Huntington’s Disease

Genomics of Huntington Disease

The gene for Huntington disease (HD), which has been cloned, was mapped to the
short arm of chromosome 4 using linkage analysis by polymorphic DNA markers.
The mutation contains an unstable trinucleotide repeat (cytosine, adenine, and gua-
nine) within a gene in the 4p16.3 chromosome. Because the tip of chromosome 4
contains 50–100 genes, It has not yet been possible to precisely localize the HD
gene. Nevertheless, it is known that the disease-causing mutation expands the length
of a repeated stretch of amino acid glutamine in the gene's product, the huntingtin
protein. Although this development may trigger the onset of HD, other genetic,
neurobiological, and environmental factors may also contribute to the progression
of the illness and underlying neuronal degeneration. A huntingtin-associated protein
has been identifi ed that binds to huntingtin; this binding is enhanced by an expanded
polyglutamine repeat, the length of which correlates to the age of disease onset. The
huntingtin-associated protein is enriched in the brain, suggesting a possible role for
selective brain pathology in HD development.

Personalized

cell therapy

Patient with

Parkinson disease

Induced pluripotent

stem cells (iPSCs)

Dopamine Fibroblasts

neurons

Transplantation Biopsy

Differentiation Reprogramming

© Jain PharmaBiotech

Fig. 12.3 Scheme of iPSCs for personalized cell therapy of Parkinson disease

12 Personalized Management of Neurological Disorders