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Project 8p Research Newsletter – April 2023

What happens when preliminary data from two independent model systems line up? Is it case closed, or just the beginning of a careful iterative process to determine which results are real and which results are not, i.e, false positives? In this case, one model is three-dimensional cortical brain organoids grown in the lab for nine months, and the other model is two-dimensional glutamatergic neurons grown in the lab for three weeks. As much as we want to believe initial positive results, they have to be rigorously repeated and pressure-tested. For any iPSC-derived model, it’s imperative to use multiple independent clones or models from additional 8p hero- derived samples.

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The Project 8p Foundation (Project 8p) was created in 2018 to:

  • Accelerate future treatments, not only for 8p, but potentially for other chromosome-wide diseases as well.
  • Lead with knowledge from patients. Currently, there is no cure for 8p disorders, nor is there a standard course of treatment.

The Project 8p Foundation (Project 8p) was created in 2018 to:

  • Raise transformative funding for pioneering scientific research into treatments for a complex, rare disease involving 250+ affected genes on the short arm of the 8 th chromosome (8p). Rearrangements of these genes causes significant abnormalities to the entire neurological system, thus all organs and functions of the body– with variance in cognitive functions, gross motor skills, social development and other challenges during infancy, and throughout life;
  • Empower a unified community of 8p patients and their families so they can have meaningful lives today; and
  • Accelerate future treatments, not only for 8p, but potentially for other chromosome-wide diseases as well.