RNU4-2 / ReNU Syndrome in the News

2025

  • Girl, 9, has rare condition diagnosed thanks to chance discovery by biologists

    itv NEWS | February 21, 2025

    The family of a young girl from Oxfordshire, whose condition was baffling doctors, say they've finally got some answers thanks to a chance discovery by biologists.

    This finding was huge and it was really exciting and unexpected that researchers were able to find a genetic cause for a genetic condition that affects thousands of families worldwide.

  • My son Charlie — and the breakthrough that changed our lives

    The Sunday Times | March 2, 2025

    James Coney and his wife, Sarah, struggled not knowing why their 12-year-old was born with a severe learning disability. In their darkest moments, they blamed themselves. Then, out of the blue, came a video call and an answer

    Knowing that it’s not your fault is really important. It’s not often enough taken into account. It just nags away in the back of your mind.

2024

  • Hundreds of thousands could be affected by newly discovered genetic disorder

    GloucestershireLive | July 12, 2024

    A new groundbreaking study of the human genome may have identified the cause of a severe disability in children and adults that is estimated to affect hundreds of thousands of people globally

    The study, which was led by Genomics England and NHS England, discovered that dozens of the patients enrolled in the landmark 100,000 Genomes Project, all had mutations in the same gene, RNU4-2 but interestingly, this particular gene has no connection to making proteins.”

  • New discovery renews hope for thousands with neurodevelopment disorders

    University of Oxford, Department of Paediatrics | July 12, 2024

    A global collaboration led by scientists at the University of Oxford has discovered that genetic variants in a specific gene cause a rare neurodevelopmental disorder (NDD).

    The study estimates that these specific changes in the RNU4-2 gene can explain 0.4% of all NDD cases globally, potentially impacting hundreds of thousands of families across the world.”

  • Scientists uncover genetic disorder that may affect thousands around world

    The Guardian | July 11, 2024

    Mutation in RNU4-2 gene linked to severe developmental delay, with hundreds of people already diagnosed

    We like to refer to RNU4-2 as “renew”, as our family is being renewed by this new information and hope for the future.”

  • Noncoding Gene Identified as Cause of Intellectual Disability Affecting Thousands

    Genetic Engineering & Biotechnology News | June 3, 2024

    Researchers at the Icahn School of Medicine at Mount Sinai, and collaborators at the University of Bristol, KU Leuven, and the NIHR BioResource, have identified a neurodevelopmental disorder, caused by mutations in a single gene, that affects tens of thousands of people worldwide.

    Analysis of national genomic diagnostic data showed RNU4-2 to be a more common etiological gene for neurodevelopmental abnormality than any previously reported autosomal gene…”

  • Researchers find a single, surprising gene behind a disorder that causes intellectual disability

    Medical Xpress | June 2, 2024

    Scientists have found the genetic root of a disorder that causes intellectual disability, which they estimate affects as many as one in 20,000 young people. And they hope their discovery leads to a new diagnosis that can provide answers to families.

    Researchers said the mutations occurred in a small "non-coding" gene, meaning it doesn't provide instructions for making proteins.

  • Researchers Identify a Genetic Cause of Intellectual Disability Affecting Tens of Thousands

    Mount Sinai Hospital | June 1, 2024

    Novel study links genetic changes in a non-coding gene called RNU4-2 to neurodevelopmental disorders

    The genetic changes we found affect a very short gene, only 141 units long, but this gene plays a crucial role in a basic biological function of cells, called gene splicing…”