Pathogenic variants in


Journal

Journal of medical genetics
ISSN: 1468-6244
Titre abrégé: J Med Genet
Pays: England
ID NLM: 2985087R

Informations de publication

Date de publication:
10 2020
Historique:
received: 12 09 2019
revised: 23 01 2020
accepted: 24 01 2020
pubmed: 11 3 2020
medline: 9 7 2021
entrez: 11 3 2020
Statut: ppublish

Résumé

Rare variants in hundreds of genes have been implicated in developmental delay (DD), intellectual disability (ID) and neurobehavioural phenotypes. Clinical and molecular characterisation was performed on 17 patients with Clinical findings included DD/ID (17/17) (speech delay in 94% (16/17), fine motor delay in 82% (14/17) and gross motor delay in 71% (12/17) of subjects), autism or autistic traits (13/17), attention deficit and hyperactivity disorder (ADHD) (11/17), other behavioural problems (7/17) and musculoskeletal findings (12/17). Other congenital malformations or clinical findings were occasionally documented. The majority of patients exhibited some dysmorphic features but no recognisable gestalt was identified. 17 heterozygous Variants in

Sections du résumé

BACKGROUND
Rare variants in hundreds of genes have been implicated in developmental delay (DD), intellectual disability (ID) and neurobehavioural phenotypes.
METHODS
Clinical and molecular characterisation was performed on 17 patients with
RESULTS
Clinical findings included DD/ID (17/17) (speech delay in 94% (16/17), fine motor delay in 82% (14/17) and gross motor delay in 71% (12/17) of subjects), autism or autistic traits (13/17), attention deficit and hyperactivity disorder (ADHD) (11/17), other behavioural problems (7/17) and musculoskeletal findings (12/17). Other congenital malformations or clinical findings were occasionally documented. The majority of patients exhibited some dysmorphic features but no recognisable gestalt was identified. 17 heterozygous
CONCLUSIONS
Variants in

Identifiants

pubmed: 32152250
pii: jmedgenet-2019-106470
doi: 10.1136/jmedgenet-2019-106470
doi:

Substances chimiques

RNA-Binding Proteins 0
TNRC6B protein, human 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

717-724

Informations de copyright

© Author(s) (or their employer(s)) 2020. No commercial re-use. See rights and permissions. Published by BMJ.

Déclaration de conflit d'intérêts

Competing interests: KM, AT and RP are employed by GeneDx, and XW, WB, JAR receive salary support from Baylor Genetics Laboratory. Both laboratories offer extensive genetic laboratory testing, including exome sequencing and derive revenue from this activity.

Auteurs

Jorge Luis Granadillo (JL)

Department of Pediatrics, Division of Genetics and Genomic Medicine, Washington University School of Medicine in Saint Louis, Saint Louis, Missouri, USA.

Alexander P A Stegmann (A)

Department of Clinical Genetics and School for Oncology and Developmental Biology, Maastricht University, Maastricht, The Netherlands.

Hui Guo (H)

Center for Medical Genetics & Hunan Key Laboratory of Medical Genetics, School of Life Sciences, Central South University, Changsha, Hunan, China.

Kun Xia (K)

Center for Medical Genetics & Hunan Key Laboratory of Medical Genetics, School of Life Sciences, Central South University, Changsha, Hunan, China.

Brad Angle (B)

Advocate Lutheran General Hospital, Park Ridge, Illinois, USA.

Kelly Bontempo (K)

Advocate Lutheran General Hospital, Park Ridge, Illinois, USA.

Judith D Ranells (JD)

Department of Pediatrics, University of South Florida, Tampa, Florida, USA.

Patricia Newkirk (P)

Department of Pediatrics, University of South Florida, Tampa, Florida, USA.

Carrie Costin (C)

Akron Children's Hospital, Akron, Ohio, USA.

Joleen Viront (J)

Akron Children's Hospital, Akron, Ohio, USA.

Constanze T Stumpel (CT)

Department of Clinical Genetics and School for Oncology and Developmental Biology, Maastricht University, Maastricht, The Netherlands.

Margje Sinnema (M)

Department of Clinical Genetics and School for Oncology and Developmental Biology, Maastricht University, Maastricht, The Netherlands.

Bianca Panis (B)

Zuyderland Medical Centre Heerlen, Heerlen, The Netherlands.

Rolph Pfundt (R)

Department of Human Genetics, Radboud University Medical Centre, Nijmgen, The Netherlands.

Ingrid P C Krapels (IPC)

Department of Clinical Genetics, Maastricht University, Maastricht, The Netherlands.

Merel Klaassens (M)

Department of Pediatrics, Maastricht University Medical Center, Maastricht, The Netherlands.

Joost Nicolai (J)

Department of Neurology, Maastricht University Medical Center, Maastricht, Netherlands.

Jinliang Li (J)

Peking University First Hospital, Beijing, Beijing, China.

Yuwu Jiang (Y)

Peking University First Hospital, Beijing, Beijing, China.

Elysa Marco (E)

UCSF Pediatric Brain Center, UCSF, San Francisco, California, USA.

Ana Canton (A)

Unidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular, Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.

Ana Claudia Latronico (AC)

Unidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular, Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.

Luciana Montenegro (L)

Unidade de Endocrinologia do Desenvolvimento, Laboratório de Hormônios e Genética Molecular, Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil.

Bruno Leheup (B)

Service de Génétique clinique, Höpital Brabois, Centre Hospitalier Universitaire de Nancy, Nancy, Lorraine, France.

Celine Bonnet (C)

Centre Hospitalier Universitaire de Nancy, Nancy, Lorraine, France.

Shivarajan M Amudhavalli (S)

Children's Mercy Hospital, Kansas City, Missouri, USA.

Caitlin E Lawson (CE)

Children's Mercy Hospital, Kansas City, Missouri, USA.

Kirsty McWalter (K)

GeneDx, Gaithersburg, Maryland, USA.

Aida Telegrafi (A)

GeneDx, Gaithersburg, Maryland, USA.

Richard Pearson (R)

GeneDx, Gaithersburg, Maryland, USA.

Malin Kvarnung (M)

Department of Clinical Genetics & Department of Molecular Medicine and Surgery, Karolinska University Hospital & Karolinska Institute, Stockholm, Sweden.

Xia Wang (X)

Baylor Genetics Laboratories, Houston, Texas, USA.

Weimin Bi (W)

Baylor College of Medicine Department of Molecular and Human Genetics, Houston, Texas, USA.

Jill Anne Rosenfeld (JA)

Baylor Genetics Laboratories, Houston, Texas, USA.
Baylor College of Medicine Department of Molecular and Human Genetics, Houston, Texas, USA.

Marwan Shinawi (M)

Department of Pediatrics, Division of Genetics and Genomic Medicine, Washington University School of Medicine in Saint Louis, Saint Louis, Missouri, USA Mshinawi@wustl.edu.

Articles similaires

[Redispensing of expensive oral anticancer medicines: a practical application].

Lisanne N van Merendonk, Kübra Akgöl, Bastiaan Nuijen
1.00
Humans Antineoplastic Agents Administration, Oral Drug Costs Counterfeit Drugs

Smoking Cessation and Incident Cardiovascular Disease.

Jun Hwan Cho, Seung Yong Shin, Hoseob Kim et al.
1.00
Humans Male Smoking Cessation Cardiovascular Diseases Female
Humans United States Aged Cross-Sectional Studies Medicare Part C
1.00
Humans Yoga Low Back Pain Female Male

Classifications MeSH