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@INPROCEEDINGS{Ruiz:283113,
author = {Ruiz, Agustin and García-González, Pablo and Puerta,
Raquel and Cano, Amanda and Olivé, Clàudia and Marquié,
Marta and Valero, Sergi and Rosende-Roca, Maitee and Sanz,
Pilar and Alegret, Montserrat and Brosseron, Frederic and
Martino-Adami, Pamela and de Rojas, Itziar and Heneka,
Michael T. and Ramirez, Alfredo and Navarro, Arcadi and
Sáez, María Eugenia and Tárraga, Lluís and Cavazos,
José E. and Boada, Mercè and Fernández, Victoria and
Socorro, Alfredo Cabrera},
title = {{L}ong‐{R}ead {S}equencing {R}eveals {A}ncestral
intragenic {APOE} {H}aplotypes with {D}istinct {R}oles in
{A}lzheimer’s {D}isease},
journal = {Alzheimer's and dementia},
volume = {21},
number = {S7},
issn = {1552-5260},
reportid = {DZNE-2026-00009},
pages = {e108005},
year = {2025},
abstract = {Background:The apolipoprotein E (APOE) ε4 allele remains
the strongest genetic risk factor for late-onset
Alzheimer’s disease (AD), yet the marked variability in
its pathogenicity suggests underlying genetic complexity.
Historically, efforts to resolve the intragenic architecture
of APOE have been hampered by the limitations of
conventional genotyping and short-read sequencing, as well
as the presence of homoplasy in common intragenic
markers—misleading similarities arising from convergent
variants.Objective:We leveraged Oxford Nanopore Technology
(ONT) to phase intragenic APOE variants, resolve homoplasy,
and examine the impact of phased haplotypes on cerebrospinal
fluid (CSF) APOE protein levels and AD
progression.Methods:Using long-read sequencing in a Spanish
memory clinic cohort (n = 1,267), we reconstructed
full-length 4 kb APOE haplotypes, identifying 59 unique
configurations grouped into five major haplogroups. Common
intragenic variants defined ancestral ε4 (4A, 4B) and ε3
(3A, 3B) haplogroups. These were analyzed for associations
with CSF APOE levels (Olink platform) and progression from
mild cognitive impairment (MCI) to dementia using adjusted
Cox regression models.Results:ONT sequencing successfully
resolved homoplasy between the APOE promoter
region—particularly at rs405509—and the canonical
protein isoforms, uncovering common but functionally
distinct ε3A/B and ε4A/B intragenic sub-haplotypes with
independent biological effects. Carriers of the ε4A
haplotype exhibited significantly lower CSF APOE protein
levels (p = 0.004), whereas the ε3B haplotype was
associated with elevated CSF APOE protein levels (p =
0.025). Notably, both haplotypes were linked to a slower
progression from MCI to AD, independent of APOE genotype,
age, sex and core CSF biomarkers.Conclusion:This study
redefines the human APOE ε3 and ε4 alleles as genetically
heterogeneous entities. Using ONT long-read sequencing, we
achieved high-resolution mapping of intragenic haplotypic
structure and regulatory variation previously obscured by
conventional approaches. This enabled the identification of
ancestral haplotypes with distinct functional profiles and
potential relevance to Alzheimer’s disease pathogenesis.
These findings highlight the importance of incorporating
haplotype-level resolution into Alzheimer’s risk
assessment, therapeutic targeting, and precision medicine
strategies.},
month = {Jul},
date = {2025-07-27},
organization = {Alzheimer’s Association
International Conference, Toronto
(Canada), 27 Jul 2025 - 31 Jul 2025},
cin = {AG Heneka},
ddc = {610},
cid = {I:(DE-2719)1011303},
pnm = {353 - Clinical and Health Care Research (POF4-353)},
pid = {G:(DE-HGF)POF4-353},
typ = {PUB:(DE-HGF)1 / PUB:(DE-HGF)16},
doi = {10.1002/alz70861_108005},
url = {https://pub.dzne.de/record/283113},
}