TY - JOUR
T1 - Increased frequency of repeat expansion mutations across different populations
AU - Ibañez, Kristina
AU - Jadhav, Bharati
AU - Zanovello, Matteo
AU - Gagliardi, Delia
AU - Clarkson, Christopher
AU - Facchini, Stefano
AU - Garg, Paras
AU - Martin-Trujillo, Alejandro
AU - Gies, Scott J.
AU - Galassi Deforie, Valentina
AU - Dalmia, Anupriya
AU - Hensman Moss, Davina J.
AU - Vandrovcova, Jana
AU - Rocca, Clarissa
AU - Moutsianas, Loukas
AU - Marini-Bettolo, Chiara
AU - Walker, Helen
AU - Turner, Chris
AU - Shoai, Maryam
AU - Long, Jeffrey D.
AU - Fratta, Pietro
AU - Langbehn, Douglas R.
AU - Tabrizi, Sarah J.
AU - Caulfield, Mark J.
AU - Cortese, Andrea
AU - Escott-Price, Valentina
AU - Hardy, John
AU - Houlden, Henry
AU - Sharp, Andrew J.
AU - Tucci, Arianna
N1 - Publisher Copyright:
© The Author(s) 2024.
PY - 2024/11
Y1 - 2024/11
N2 - Repeat expansion disorders (REDs) are a devastating group of predominantly neurological diseases. Together they are common, affecting 1 in 3,000 people worldwide with population-specific differences. However, prevalence estimates of REDs are hampered by heterogeneous clinical presentation, variable geographic distributions and technological limitations leading to underascertainment. Here, leveraging whole-genome sequencing data from 82,176 individuals from different populations, we found an overall disease allele frequency of REDs of 1 in 283 individuals. Modeling disease prevalence using genetic data, age at onset and survival, we show that the expected number of people with REDs would be two to three times higher than currently reported figures, indicating underdiagnosis and/or incomplete penetrance. While some REDs are population specific, for example, Huntington disease-like 2 in Africans, most REDs are represented in all broad genetic ancestries (that is, Europeans, Africans, Americans, East Asians and South Asians), challenging the notion that some REDs are found only in specific populations. These results have worldwide implications for local and global health communities in the diagnosis and counseling of REDs.
AB - Repeat expansion disorders (REDs) are a devastating group of predominantly neurological diseases. Together they are common, affecting 1 in 3,000 people worldwide with population-specific differences. However, prevalence estimates of REDs are hampered by heterogeneous clinical presentation, variable geographic distributions and technological limitations leading to underascertainment. Here, leveraging whole-genome sequencing data from 82,176 individuals from different populations, we found an overall disease allele frequency of REDs of 1 in 283 individuals. Modeling disease prevalence using genetic data, age at onset and survival, we show that the expected number of people with REDs would be two to three times higher than currently reported figures, indicating underdiagnosis and/or incomplete penetrance. While some REDs are population specific, for example, Huntington disease-like 2 in Africans, most REDs are represented in all broad genetic ancestries (that is, Europeans, Africans, Americans, East Asians and South Asians), challenging the notion that some REDs are found only in specific populations. These results have worldwide implications for local and global health communities in the diagnosis and counseling of REDs.
UR - http://www.scopus.com/inward/record.url?scp=85205544339&partnerID=8YFLogxK
U2 - 10.1038/s41591-024-03190-5
DO - 10.1038/s41591-024-03190-5
M3 - Article
C2 - 39354197
AN - SCOPUS:85205544339
SN - 1078-8956
VL - 30
SP - 3357
EP - 3368
JO - Nature Medicine
JF - Nature Medicine
IS - 11
ER -