Clinical application of next-generation sequencing to the practice of neurology

被引:82
作者
Rexach, Jessica [1 ]
Lee, Hane [2 ,3 ]
Martinez-Agosto, Julian A. [3 ,4 ]
Nemeth, Andrea H. [6 ,7 ]
Fogel, Brent L. [1 ,3 ,5 ]
机构
[1] Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Program Neurogenet, Los Angeles, CA 90095 USA
[2] Univ Calif Los Angeles, David Geffen Sch Med, Dept Pathol & Lab Med, Los Angeles, CA 90095 USA
[3] Univ Calif Los Angeles, David Geffen Sch Med, Dept Human Genet, Los Angeles, CA 90095 USA
[4] Univ Calif Los Angeles, David Geffen Sch Med, Dept Pediat, Div Med Genet, Los Angeles, CA 90095 USA
[5] Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Clin Neurogen Res Ctr, Los Angeles, CA 90095 USA
[6] Univ Oxford, Nuffield Dept Clin Neurosci, Oxford, England
[7] Oxford Univ Hosp Natl Hlth Serv Fdn Trust, Oxford Ctr Genom Med, Oxford, England
关键词
HETEROZYGOUS HTRA1 MUTATIONS; ALZHEIMERS-DISEASE; PRECISION MEDICINE; NEURODEGENERATIVE DISEASES; MOLECULAR DIAGNOSIS; NATIONAL INSTITUTE; GENETIC-BASIS; ENCODE DATA; EXOME; DISORDERS;
D O I
10.1016/S1474-4422(19)30033-X
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Next-generation sequencing technologies allow for rapid and inexpensive large-scale genomic analysis, creating unprecedented opportunities to integrate genomic data into the clinical diagnosis and management of neurological disorders. However, the scale and complexity of these data make them difficult to interpret and require the use of sophisticated bioinformatics applied to extensive datasets, including whole exome and genome sequences. Detailed analysis of genetic data has shown that accurate phenotype information is essential for correct interpretation of genetic variants and might necessitate re-evaluation of the patient in some cases. A multidisciplinary approach that incorporates bioinformatics, clinical evaluation, and human genetics can help to address these challenges. However, despite numerous studies that show the efficacy of next-generation sequencing in establishing molecular diagnoses, pathogenic mutations are generally identified in fewer than half of all patients with genetic neurological disorders, exposing considerable gaps in the understanding of the human genome and providing opportunities to focus research on improving the usefulness of genomics in clinical practice. Looking forward, the emergence of precision health in neurological care will increasingly apply genomic data analysis to pharmacogenetics, preventive medicine, and patient-targeted therapies.
引用
收藏
页码:492 / 503
页数:12
相关论文
共 149 条
[1]   Genetic identification of familial hypercholesterolemia within a single US health care system [J].
Abul-Husn, Noura S. ;
Manickam, Kandamurugu ;
Jones, Laney K. ;
Wright, Eric A. ;
Hartzel, Dustin N. ;
Gonzaga-Jauregui, Claudia ;
O'Dushlaine, Colm ;
Leader, Joseph B. ;
Kirchner, H. Lester ;
Lindbuchler, D'Andra M. ;
Barr, Marci L. ;
Giovanni, Monica A. ;
Ritchie, Marylyn D. ;
Overton, John D. ;
Reid, Jeffrey G. ;
Metpally, Raghu P. R. ;
Wardeh, Amr H. ;
Borecki, Ingrid B. ;
Yancopoulos, George D. ;
Baras, Aris ;
Shuldiner, Alan R. ;
Gottesman, Omri ;
Ledbetter, David H. ;
Carey, David J. ;
Dewey, Frederick E. ;
Murray, Michael F. .
SCIENCE, 2016, 354 (6319)
[2]   New insights into the generation and role of de novo mutations in health and disease [J].
Acuna-Hidalgo, Rocio ;
Veltman, Joris A. ;
Hoischen, Alexander .
GENOME BIOLOGY, 2016, 17
[3]   The PsychENCODE project [J].
Akbarian, Schahram ;
Liu, Chunyu ;
Knowles, James A. ;
Vaccarino, Flora M. ;
Farnham, Peggy J. ;
Crawford, Gregory E. ;
Jaffe, Andrew E. ;
Pinto, Dalila ;
Dracheva, Stella ;
Geschwind, Daniel H. ;
Mill, Jonathan ;
Nairn, Angus C. ;
Abyzov, Alexej ;
Pochareddy, Sirisha ;
Prabhakar, Shyam ;
Weissman, Sherman ;
Sullivan, Patrick F. ;
State, Matthew W. ;
Weng, Zhiping ;
Peters, Mette A. ;
White, Kevin P. ;
Gerstein, Mark B. ;
Amiri, Anahita ;
Armoskus, Chris ;
Ashley-Koch, Allison E. ;
Bae, Taejeong ;
Beckel-Mitchener, Andrea ;
Berman, Benjamin P. ;
Coetzee, Gerhard A. ;
Coppola, Gianfilippo ;
Francoeur, Nancy ;
Fromer, Menachem ;
Gao, Robert ;
Grennan, Kay ;
Herstein, Jennifer ;
Kavanagh, David H. ;
Ivanov, Nikolay A. ;
Jiang, Yan ;
Kitchen, Robert R. ;
Kozlenkov, Alexey ;
Kundakovic, Marija ;
Li, Mingfeng ;
Li, Zhen ;
Liu, Shuang ;
Mangravite, Lara M. ;
Mattei, Eugenio ;
Markenscoff-Papadimitriou, Eirene ;
Navarro, Fabio C. P. ;
North, Nicole ;
Omberg, Larsson .
NATURE NEUROSCIENCE, 2015, 18 (12) :1707-1712
[4]   Mutations in CSPP1 Lead to Classical Joubert Syndrome [J].
Akizu, Naiara ;
Silhavy, Jennifer L. ;
Rosti, Rasim Ozgur ;
Scott, Eric ;
Fenstermaker, Ali G. ;
Schroth, Jana ;
Zaki, Maha S. ;
Sanchez, Henry ;
Gupta, Neerja ;
Kabra, Madhulika ;
Kara, Majdi ;
Ben-Omran, Tawfeg ;
Rosti, Basak ;
Guemez-Gamboa, Alicia ;
Spencer, Emily ;
Pan, Roger ;
Cai, Na ;
Abdellateef, Mostafa ;
Gabriel, Stacey ;
Halbritter, Jan ;
Hildebrandt, Friedhelm ;
van Bokhoven, Hans ;
Gunel, Murat ;
Gleeson, Joseph G. .
AMERICAN JOURNAL OF HUMAN GENETICS, 2014, 94 (01) :80-86
[5]   Whole-genome sequencing offers additional but limited clinical utility compared with reanalysis of whole-exome sequencing [J].
Alfares, Ahmed ;
Aloraini, Taghrid ;
Al Subaie, Lamia ;
Alissa, Abdulelah ;
Al Qudsi, Ahmed ;
Alahmad, Ahmed ;
Al Mutairi, Fuad ;
Alswaid, Abdulrahman ;
Alothaim, Ali ;
Eyaid, Wafaa ;
Albalwi, Mohammed ;
Alturki, Saeed ;
Alfadhel, Majid .
GENETICS IN MEDICINE, 2018, 20 (11) :1328-1333
[6]   A global reference for human genetic variation [J].
Altshuler, David M. ;
Durbin, Richard M. ;
Abecasis, Goncalo R. ;
Bentley, David R. ;
Chakravarti, Aravinda ;
Clark, Andrew G. ;
Donnelly, Peter ;
Eichler, Evan E. ;
Flicek, Paul ;
Gabriel, Stacey B. ;
Gibbs, Richard A. ;
Green, Eric D. ;
Hurles, Matthew E. ;
Knoppers, Bartha M. ;
Korbel, Jan O. ;
Lander, Eric S. ;
Lee, Charles ;
Lehrach, Hans ;
Mardis, Elaine R. ;
Marth, Gabor T. ;
McVean, Gil A. ;
Nickerson, Deborah A. ;
Wang, Jun ;
Wilson, Richard K. ;
Boerwinkle, Eric ;
Doddapaneni, Harsha ;
Han, Yi ;
Korchina, Viktoriya ;
Kovar, Christie ;
Lee, Sandra ;
Muzny, Donna ;
Reid, Jeffrey G. ;
Zhu, Yiming ;
Chang, Yuqi ;
Feng, Qiang ;
Fang, Xiaodong ;
Guo, Xiaosen ;
Jian, Min ;
Jiang, Hui ;
Jin, Xin ;
Lan, Tianming ;
Li, Guoqing ;
Li, Jingxiang ;
Li, Yingrui ;
Liu, Shengmao ;
Liu, Xiao ;
Lu, Yao ;
Ma, Xuedi ;
Tang, Meifang ;
Wang, Bo .
NATURE, 2015, 526 (7571) :68-+
[7]   Performance of ACMG-AMP Variant-Interpretation Guidelines among Nine Laboratories in the Clinical Sequencing Exploratory Research Consortium (vol 98, pg 1067, 2016) [J].
Amendola, Laura M. ;
Jarvik, Gail P. ;
Leo, Michael C. ;
McLaughlin, Heather M. ;
Akkari, Yassmine ;
Amaral, Michelle D. ;
Berg, Jonathan S. ;
Biswas, Sawona ;
Bowling, Kevin M. ;
Conlin, Laura K. ;
Cooper, Greg M. ;
Dorschner, Michael O. ;
Dulik, Matthew C. ;
Ghazani, Arezou A. ;
Ghosh, Rajarshi ;
Green, Robert C. ;
Hart, Ragan ;
Horton, Carrie ;
Johnston, Jennifer J. ;
Lebo, Matthew S. ;
Milosavljevic, Aleksandar ;
Ou, Jeffrey ;
Pak, Christine M. ;
Patel, Ronak Y. ;
Punj, Sumit ;
Richards, Carolyn Sue ;
Salama, Joseph ;
Strande, Natasha T. ;
Yang, Yaping ;
Plon, Sharon E. ;
Biesecker, Leslie G. ;
Rehm, Heidi L. .
AMERICAN JOURNAL OF HUMAN GENETICS, 2016, 99 (01) :247-247
[8]   E pluribus unum [J].
不详 .
NATURE METHODS, 2010, 7 (05) :331-331
[9]   The Genotype-Tissue Expression (GTEx) pilot analysis: Multitissue gene regulation in humans [J].
Ardlie, Kristin G. ;
DeLuca, David S. ;
Segre, Ayellet V. ;
Sullivan, Timothy J. ;
Young, Taylor R. ;
Gelfand, Ellen T. ;
Trowbridge, Casandra A. ;
Maller, Julian B. ;
Tukiainen, Taru ;
Lek, Monkol ;
Ward, Lucas D. ;
Kheradpour, Pouya ;
Iriarte, Benjamin ;
Meng, Yan ;
Palmer, Cameron D. ;
Esko, Tonu ;
Winckler, Wendy ;
Hirschhorn, Joel N. ;
Kellis, Manolis ;
MacArthur, Daniel G. ;
Getz, Gad ;
Shabalin, Andrey A. ;
Li, Gen ;
Zhou, Yi-Hui ;
Nobel, Andrew B. ;
Rusyn, Ivan ;
Wright, Fred A. ;
Lappalainen, Tuuli ;
Ferreira, Pedro G. ;
Ongen, Halit ;
Rivas, Manuel A. ;
Battle, Alexis ;
Mostafavi, Sara ;
Monlong, Jean ;
Sammeth, Michael ;
Mele, Marta ;
Reverter, Ferran ;
Goldmann, Jakob M. ;
Koller, Daphne ;
Guigo, Roderic ;
McCarthy, Mark I. ;
Dermitzakis, Emmanouil T. ;
Gamazon, Eric R. ;
Im, Hae Kyung ;
Konkashbaev, Anuar ;
Nicolae, Dan L. ;
Cox, Nancy J. ;
Flutre, Timothee ;
Wen, Xiaoquan ;
Stephens, Matthew .
SCIENCE, 2015, 348 (6235) :648-660
[10]   Paternal germline mosaicism in collagen VI related myopathies [J].
Armaroli, Annarita ;
Trabanelli, Cecilia ;
Scotton, Chiara ;
Venturoli, Anna ;
Seluatici, Rita ;
Brisca, Giacomo ;
Merlini, Luciano ;
Bruno, Claudio ;
Ferlini, Alessandra ;
Gualandi, Francesca .
EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY, 2015, 19 (05) :533-536