Literature DB >> 35931048

Neurodevelopmental copy-number variants: A roadmap to improving outcomes by uniting patient advocates, researchers, and clinicians for collective impact.

.   

Abstract

Copy-number variants and structural variants (CNVs/SVs) drive many neurodevelopmental-related disorders. While many neurodevelopmental-related CNVs/SVs give rise to complex phenotypes, the overlap in phenotypic presentation between independent CNVs can be extensive and provides a motivation for shared approaches. This confluence at the level of clinical phenotype implies convergence in at least some aspects of the underlying genomic mechanisms. With this perspective, our Commission on Novel Technologies for Neurodevelopmental CNVs asserts that the time has arrived to approach neurodevelopmental-related CNVs/SVs as a class of disorders that can be identified, investigated, and treated on the basis of shared mechanisms and/or pathways (e.g., molecular, neurological, or developmental). To identify common etiologic mechanisms among uncommon neurodevelopmental-related disorders and to potentially identify common therapies, it is paramount for teams of scientists, clinicians, and patients to unite their efforts. We bring forward novel, collaborative, and integrative strategies to translational CNV/SV research that engages diverse stakeholders to help expedite therapeutic outcomes. We articulate a clear vision for piloted roadmap strategies to reduce patient/caregiver burden and redundancies, increase efficiency, avoid siloed data, and accelerate translational discovery across CNV/SV-based syndromes.
Copyright © 2022 American Society of Human Genetics. Published by Elsevier Inc. All rights reserved.

Entities:  

Keywords:  CNVs; biobank; community engagement; copy-number variants; genomic disorders; iPSCs; inclusion; infrastructure; long-read sequencing; neurodevelopment; neurological; patient centered; patient led; structural variants; systematic phenotyping; team science

Mesh:

Year:  2022        PMID: 35931048      PMCID: PMC9388383          DOI: 10.1016/j.ajhg.2022.07.003

Source DB:  PubMed          Journal:  Am J Hum Genet        ISSN: 0002-9297            Impact factor:   11.043


  68 in total

1.  Complex Oscillatory Waves Emerging from Cortical Organoids Model Early Human Brain Network Development.

Authors:  Cleber A Trujillo; Richard Gao; Priscilla D Negraes; Jing Gu; Justin Buchanan; Sebastian Preissl; Allen Wang; Wei Wu; Gabriel G Haddad; Isaac A Chaim; Alain Domissy; Matthieu Vandenberghe; Anna Devor; Gene W Yeo; Bradley Voytek; Alysson R Muotri
Journal:  Cell Stem Cell       Date:  2019-08-29       Impact factor: 24.633

Review 2.  Recurrent reciprocal copy number variants: Roles and rules in neurodevelopmental disorders.

Authors:  Aditi Deshpande; Lauren A Weiss
Journal:  Dev Neurobiol       Date:  2018-03-25       Impact factor: 3.964

3.  Small dsRNAs induce transcriptional activation in human cells.

Authors:  Long-Cheng Li; Steven T Okino; Hong Zhao; Deepa Pookot; Robert F Place; Shinji Urakami; Hideki Enokida; Rajvir Dahiya
Journal:  Proc Natl Acad Sci U S A       Date:  2006-11-03       Impact factor: 11.205

4.  The ring 14 syndrome: clinical and molecular definition.

Authors:  Marcella Zollino; Laura Seminara; Daniela Orteschi; Giuseppe Gobbi; Simona Giovannini; Elvio Della Giustina; Daniele Frattini; Angela Scarano; Giovanni Neri
Journal:  Am J Med Genet A       Date:  2009-06       Impact factor: 2.802

5.  Cell Painting, a high-content image-based assay for morphological profiling using multiplexed fluorescent dyes.

Authors:  Mark-Anthony Bray; Shantanu Singh; Han Han; Chadwick T Davis; Blake Borgeson; Cathy Hartland; Maria Kost-Alimova; Sigrun M Gustafsdottir; Christopher C Gibson; Anne E Carpenter
Journal:  Nat Protoc       Date:  2016-08-25       Impact factor: 13.491

6.  DECIPHER: Database of Chromosomal Imbalance and Phenotype in Humans Using Ensembl Resources.

Authors:  Helen V Firth; Shola M Richards; A Paul Bevan; Stephen Clayton; Manuel Corpas; Diana Rajan; Steven Van Vooren; Yves Moreau; Roger M Pettett; Nigel P Carter
Journal:  Am J Hum Genet       Date:  2009-04-02       Impact factor: 11.025

7.  Generation of human brain region-specific organoids using a miniaturized spinning bioreactor.

Authors:  Xuyu Qian; Fadi Jacob; Mingxi Max Song; Ha Nam Nguyen; Hongjun Song; Guo-Li Ming
Journal:  Nat Protoc       Date:  2018-02-22       Impact factor: 13.491

8.  Organoid single-cell genomic atlas uncovers human-specific features of brain development.

Authors:  Sabina Kanton; Michael James Boyle; Zhisong He; Malgorzata Santel; Anne Weigert; Fátima Sanchís-Calleja; Patricia Guijarro; Leila Sidow; Jonas Simon Fleck; Dingding Han; Zhengzong Qian; Michael Heide; Wieland B Huttner; Philipp Khaitovich; Svante Pääbo; Barbara Treutlein; J Gray Camp
Journal:  Nature       Date:  2019-10-16       Impact factor: 49.962

9.  Human Ring Chromosomes - New Insights for their Clinical Significance.

Authors:  Rs Guilherme; E Klein; Ab Hamid; S Bhatt; M Volleth; A Polityko; A Kulpanovich; A Dufke; B Albrecht; S Morlot; L Brecevic; Mb Petersen; E Manolakos; N Kosyakova; T Liehr
Journal:  Balkan J Med Genet       Date:  2013-06       Impact factor: 0.519

10.  The mutational constraint spectrum quantified from variation in 141,456 humans.

Authors:  Konrad J Karczewski; Laurent C Francioli; Grace Tiao; Beryl B Cummings; Jessica Alföldi; Qingbo Wang; Ryan L Collins; Kristen M Laricchia; Andrea Ganna; Daniel P Birnbaum; Laura D Gauthier; Harrison Brand; Matthew Solomonson; Nicholas A Watts; Daniel Rhodes; Moriel Singer-Berk; Eleina M England; Eleanor G Seaby; Jack A Kosmicki; Raymond K Walters; Katherine Tashman; Yossi Farjoun; Eric Banks; Timothy Poterba; Arcturus Wang; Cotton Seed; Nicola Whiffin; Jessica X Chong; Kaitlin E Samocha; Emma Pierce-Hoffman; Zachary Zappala; Anne H O'Donnell-Luria; Eric Vallabh Minikel; Ben Weisburd; Monkol Lek; James S Ware; Christopher Vittal; Irina M Armean; Louis Bergelson; Kristian Cibulskis; Kristen M Connolly; Miguel Covarrubias; Stacey Donnelly; Steven Ferriera; Stacey Gabriel; Jeff Gentry; Namrata Gupta; Thibault Jeandet; Diane Kaplan; Christopher Llanwarne; Ruchi Munshi; Sam Novod; Nikelle Petrillo; David Roazen; Valentin Ruano-Rubio; Andrea Saltzman; Molly Schleicher; Jose Soto; Kathleen Tibbetts; Charlotte Tolonen; Gordon Wade; Michael E Talkowski; Benjamin M Neale; Mark J Daly; Daniel G MacArthur
Journal:  Nature       Date:  2020-05-27       Impact factor: 69.504

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.