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What is the Future of Stem Cell Research in ASD?

Induced pluripotent stem cells (iPSCs) give us the opportunity to examine the shape and function of a human neuron, a cell type that is extremely difficult to study in living humans. We now have the ability to determine why some genetic variability has more severe effects than other genetic variability by generating stem cells from different patients and studying neuron development as it is happening.

Click here to learn how iPSCs can be used to screen drugs.


A vision for the future

One of the ultimate goals in all ASD research is to establish a mechanism to individualize diagnosis and therapy. The biological basis of disorders along the ASD spectrum can be very different; each individual may require a unique type of treatment.

With the use of stem cell technology, it is quite possible that, one day, patients who exhibit behaviors that fall within the ASD criteria will be able to walk into a doctor’s office, give a skin biopsy sample, learn in a matter of weeks about a possible underlying cause of their disorder, and receive advice about a targeted therapy.

Induced pluripotent stem cells are not necessarily a silver bullet to overcome all ASD research barriers, but they are a giant leap forward. Similarly, future scenarios like the one described above may not apply to all ASD patients, given that ASD is linked to both genetic and environmental factors. But the information we stand to gain through iPSC studies can open new doors to address the challenges of ASD diagnosis and therapy.

  1. Marchetto MC, et al. (2010) A model for neural development and treatment of Rett Syndrome using human induced pluripotent stem cells. Cell 143:527-539. PMID: 21074045.
  2. Liu Y, Rao M (2011) Gene targeting in human pluripotent stem cells. Methods Mol Biol 767:355-367. PMID: 21822888.
  3. Rastegar M, et al. (2009) MECP2 isoform-specific vectors with regulated expression for Rett Syndrome gene therapy. PLoS One4(8):e6810. PMID: 19710912.