Suppressing Aneuploidy-associated phenotypes in Down syndrome

NIH RePORTER · NIH · R01 · $1,720,291 · view on reporter.nih.gov ↗

Abstract

Project Summary An abnormal number of chromosomes or aneuploidy accounts for most spontaneous abortions as missegregation of a single chromosome during development is often lethal.Patients with trisomies for chromosomes 13 or 18, which cause Patau and Edwards syndromes, respectively, are born with severe developmental defects and die soon after birth. Only patients with trisomy 21, which causes Down syndrome can live to adulthoodbut show cognitive disabilities, increased risk for leukemias, autoimmune disorders, and clinical symptoms associated with premature aging. Importantly, the incidence of aneuploidy increases with age in both somatic and germline tissues in apparently healthy individuals. The mechanisms by which aneuploidy affects cellular functionto cause Down syndrome or promote aging are not understood. Our preliminary data reveal that aneuploidy disrupts the integrity and morphology of the nuclear membrane. Because mutations that affect nuclear morphology cause premature aging, we hypothesize that the aneuploidy effects on the nucleus drive phenotypic anomalies associated with premature aging in Down syndrome. Here, we plan to identify the mechanisms through which aneuploidy affects the nucleus, to investigate how an abnormal nucleus contributes to the pathophysiology of trisomy 21, and to target biochemical pathways so as to suppress aneuploidy-associated phenotypes in trisomy 21 cells.

Key facts

NIH application ID
10536927
Project number
1R01HD107873-01A1
Recipient
UNIV OF MASSACHUSETTS MED SCH WORCESTER
Principal Investigator
Eduardo Martin Torres
Activity code
R01
Funding institute
NIH
Fiscal year
2022
Award amount
$1,720,291
Award type
1
Project period
2022-09-01 → 2025-08-31