IPSC Models of Chromosome 15Q Imprinting Disorders: From Disease Modeling to Therapeutic Strategies

Adv Neurobiol. 2020:25:55-77. doi: 10.1007/978-3-030-45493-7_3.

Abstract

The chromosome 15q11-q13 region of the human genome is regulated by genomic imprinting, an epigenetic phenomenon in which genes are expressed exclusively from one parental allele. Several genes within the 15q11-q13 region are expressed exclusively from the paternally inherited chromosome 15. At least one gene UBE3A, shows exclusive expression of the maternal allele, but this allele-specific expression is restricted to neurons. The appropriate regulation of imprinted gene expression across chromosome 15q11-q13 has important implications for human disease. Three different neurodevelopmental disorders result from aberrant expression of imprinted genes in this region: Prader-Willi syndrome (PWS), Angelman syndrome (AS), and 15q duplication syndrome.

Keywords: Angelman syndrome; Antisense oligonucleotides; Chromosome 15q11-q13; Dup15q syndrome; Genomic imprinting; Prader–Willi syndrome; UBE3A.

MeSH terms

  • Angelman Syndrome* / genetics
  • Chromosomes
  • Genomic Imprinting / genetics
  • Humans
  • Prader-Willi Syndrome* / genetics