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Prospects for improving brain function in individuals with Down syndrome.

Alberto C S Costa1, Jonah J Scott-McKean

  • 1Division of Pediatric Neurology, Department of Pediatrics, Case Western Reserve School of Medicine, 11100 Euclid Avenue, Mail Stop RBC 6090, Cleveland, OH 44106, USA. alberto.costa@case.edu

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Researchers are exploring new drug therapies to improve learning and memory deficits in Down syndrome (DS). Promising preclinical studies and ongoing clinical trials offer hope for future pharmacotherapies to aid individuals with DS.

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Area of Science:

  • Neuroscience
  • Genetics
  • Pharmacology

Background:

  • Down syndrome (DS), caused by trisomy 21, is the leading genetic cause of intellectual disability.
  • Currently, no approved pharmacotherapy exists for cognitive and adaptive deficits in DS.

Purpose of the Study:

  • To review available mouse models for Down syndrome.
  • To comprehensively review preclinical research strategies targeting cognitive deficits in DS.
  • To briefly review clinical studies for DS pharmacotherapies.

Main Methods:

  • Review of preclinical research on pharmacological agents in DS mouse models.
  • Categorization of strategies including antioxidant agents, enzyme inhibitors, receptor modulators, and growth factors.
  • Summary of ongoing and concluded clinical studies in DS.

Main Results:

  • Multiple preclinical strategies show promise in rescuing learning/memory deficits in DS mouse models.
  • Ten distinct preclinical research strategies were identified and reviewed.
  • Five strategies have progressed to clinical trials in individuals with DS.

Conclusions:

  • Promising pharmacological agents are emerging for Down syndrome research.
  • Future pharmacotherapies may serve as valuable adjuncts to traditional interventions for DS.
  • Significant advancements in DS pharmacotherapy are anticipated within the next decade.