Genomics and Radiogenomics in Inherited Neurometabolic Disorders - A Practical Primer for Pediatricians

Indian J Pediatr. 2019 Oct;86(10):923-938. doi: 10.1007/s12098-019-02860-4. Epub 2019 Jun 13.

Abstract

Advances in genetics has revolutionised the way we understand, diagnose and manage neurological disorders. Notwithstanding the fact that genetic confirmation has already become standard of care in routine clinical practice, radiological and clinical phenotyping has not diminished in value; in fact it has found an enhanced role in guiding and interpreting genetic test results. Inherited neurometabolic disorders are a prominent group of disorders which are seen commonly in clinical practice and many are potentially treatable. The concept of Radiogenomics is the bridge from phenotype to genotype and the strength of association varies widely across different inherited metabolic diseases. Understanding the strengths and limitations of these correlations forms the basis of success of multidisciplinary approach to diagnose these disorders. In this article authors give a brief overview of the genetic basis of a disease, available genetic tests and the prominent role of radiology in contemplating a diagnostic suspicion and guiding further confirmatory tests.

Keywords: Genetics; Inborn errors of metabolism; Mitochondrial diseases; Neurometabolic disorders; Next generation sequencing; Radiogenomics; Whole exome sequencing.

Publication types

  • Case Reports
  • Review

MeSH terms

  • ATP Binding Cassette Transporter, Subfamily D, Member 1 / genetics
  • Adult
  • Algorithms
  • Brain Diseases, Metabolic / diagnostic imaging*
  • Brain Diseases, Metabolic / genetics
  • Child
  • Child, Preschool
  • Diagnostic Imaging / methods
  • Female
  • Genetic Diseases, Inborn / diagnosis
  • Genetic Diseases, Inborn / genetics
  • Genetic Testing / methods
  • Genomics / methods*
  • Genotype
  • Humans
  • Male
  • Membrane Proteins / genetics
  • Metabolic Diseases / diagnostic imaging*
  • Metabolic Diseases / genetics
  • Mitochondrial Proteins / genetics
  • Nervous System Diseases / diagnostic imaging*
  • Nervous System Diseases / genetics
  • Pediatricians*
  • Phenotype
  • Radiology / methods*
  • Young Adult

Substances

  • ABCD1 protein, human
  • ATP Binding Cassette Transporter, Subfamily D, Member 1
  • Membrane Proteins
  • Mitochondrial Proteins
  • Surf-1 protein