Retinoblastoma: From genes to patient care


Por: Bouchoucha, Y, Matet, A, Berger, A, Carcaboso AM, Gerrish, A, Moll, A, Jenkinson, H, Ketteler, P, Dorsmank, JC, Chantada G, Beck-Popovic, M, Munier, F, Aerts, I, Doz, F and Golmard, L

Publicada: 1 ene 2023 Ahead of Print: 1 dic 2022
Resumen:
Retinoblastoma is the most common paediatric neoplasm of the retina, and one of the earliest model of cancer genetics since the identification of the master tumour suppressor gene RB1. Tumorigenesis has been shown to be driven by pathogenic variants of the RB1 locus, but also genomic and epigenomic alterations outside the locus. The increasing knowledge on this "mutational landscape " is used in current practice for precise genetic testing and counselling. Novel methods provide access to pre-therapeutic tumour DNA, by isolating cell-free DNA from aqueous humour or plasma. This is expected to facilitate assessment of the constitutional status of RB1, to provide an early risk stratification using molecular prognostic markers, to follow the response to the treatment in longitudinal studies, and to predict the response to targeted therapies. The aim of this review is to show how molecular genetics of retinoblastoma drives diagnosis, treatment, monitoring of the disease and surveillance of the patients and relatives. We first recap the current knowledge on retinoblastoma genetics and its use in every-day practice. We then focus on retinoblastoma subgrouping at the era of molecular biology, and the expected input of cell-free DNA in the field.

Filiaciones:
Bouchoucha, Y:
 SIREDO Oncology Center of Care, Innovation and Research for Children, Adolescent and Young Adults with Cancer, Institut Curie, Paris, France

 Université Paris-Cité, Paris, France

 SIREDO Oncol Ctr Care Innovat & Res Children, Inst Curie, Adolescent & Young Adults Canc, Paris, France

 Univ Paris Cite, Paris, France

Matet, A:
 Université Paris-Cité, Paris, France

 Department of Ophthalmology, Institut Curie, Paris, France

 Univ Paris Cite, Paris, France

 Inst Curie, Dept Ophthalmol, Paris, France

Berger, A:
 Department of Ophthalmology, University of Lausanne, Jules-Gonin Eye Hospital, Fondation Asile des Aveugles, Lausanne, Switzerland

 Univ Lausanne, Jules Gonin Eye Hosp, Dept Ophthalmol, Fdn Asile Aveugles, Lausanne, Switzerland

Carcaboso AM:
 Institut de Recerca Sant Joan de Déu, Barcelona, Spain

 SJD Pediatric Cancer Center Barcelona, Hospital Sant Joan de Deu, Barcelona, 08950, Spain

 Inst Recerca Sant Joan Deu, Barcelona, Spain

 Hosp San Juan Dios, SJD Pediat Canc Ctr Barcelona, Barcelona 08950, Spain

Gerrish, A:
 West Midlands Regional Genetics Service, Birmingham Women's and Children's NHS Foundation Trust, Birmingham, B15 2TG, UK

 Birmingham Womens & Childrens NHS Fdn Trust, West Midlands Reg Genet Serv, Birmingham B15 2TG, England

Moll, A:
 Department of Ophthalmology, VU University Medical Center, Amsterdam, the Netherlands

 Vrije Univ Amsterdam Med Ctr, Dept Ophthalmol, Amsterdam, Netherlands

Jenkinson, H:
 Department of Paediatric Oncology, Birmingham Women's and Children's NHS Foundation Trust, Birmingham, B4 6NH, UK

 Birmingham Womens & Childrens NHS Fdn Trust, Dept Paediat Oncol, Birmingham B4 6NH, England

Ketteler, P:
 Department of Paediatrics III, University Hospital Essen, Essen, Germany

 Univ Hosp Essen, Dept Paediat 3, Essen, Germany

Dorsmank, JC:
 Department of Human Genetics, Amsterdam UMC location VUmc, Amsterdam, the Netherlands

 Amsterdam UMC locat VUmc, Dept Human Genet, Amsterdam, Netherlands

Chantada G:
 Institut de Recerca Sant Joan de Déu, Barcelona, Spain

 SJD Pediatric Cancer Center Barcelona, Hospital Sant Joan de Deu, Barcelona, 08950, Spain

 Inst Recerca Sant Joan Deu, Barcelona, Spain

 Hosp San Juan Dios, SJD Pediat Canc Ctr Barcelona, Barcelona 08950, Spain

Beck-Popovic, M:
 Department of Pediatrics, Hematology-Oncology Unit, University Hospital, Lausanne, Switzerland

 Univ Hosp, Dept Pediat, Hematol Oncol Unit, Lausanne, Switzerland

Munier, F:
 Unit of Pediatric Ocular Oncology, Jules-Gonin Eye Hospital, University of Lausanne, Lausanne, Switzerland

 Univ Lausanne, Jules Gonin Eye Hosp, Unit Pediat Ocular Oncol, Lausanne, Switzerland

Aerts, I:
 SIREDO Oncology Center of Care, Innovation and Research for Children, Adolescent and Young Adults with Cancer, Institut Curie, Paris, France

Doz, F:
 SIREDO Oncology Center of Care, Innovation and Research for Children, Adolescent and Young Adults with Cancer, Institut Curie, Paris, France

 Université Paris-Cité, Paris, France

 SIREDO Oncol Ctr Care Innovat & Res Children, Inst Curie, Adolescent & Young Adults Canc, Paris, France

 Univ Paris Cite, Paris, France

Golmard, L:
 Department of Genetics, PSL Research University, Institut Curie, Paris, France

 PSL Res Univ, Inst Curie, Dept Genet, Paris, France

SIREDO Oncology Center of Care, Innovation and Research for Children, Adolescent and Young Adults with Cancer, Institut Curie, Paris, France; Universite Paris-Cite, Paris, France.
ISSN: 17697212





European Journal of Medical Genetics
Editorial
ELSEVIER, RADARWEG 29, 1043 NX AMSTERDAM, NETHERLANDS, Países Bajos
Tipo de documento: Article
Volumen: 66 Número: 1
Páginas: 104674-104674
WOS Id: 000899351000010
ID de PubMed: 36470558
imagen hybrid

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