'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis

'Find a way, or make one': How two scientists hope to revitalize CRISPR's rare disease crisis

Fyodor Urnov and Kiran Musunuru advocate for streamlined CRISPR treatments for rare genetic diseases, partnering with Danaher and CHOP to develop custom therapies for HLH and metabolic disorders.

The representative of the Hrabriša association Ivana Badnjarević spoke for Euronews Serbia.,juronjuz serbia, euronews serbia, euronews serbia, euronews serbia, juronjuz, euronews

The Children’s Hospital Niño Jesús de la Comunidad de Madrid has participated in an international clinical trial with gene therapy that has cured nine children with leukocyte adhesion deficiency 1 (LAD-1), a serious rare disease. In this case, the beneficiary has been a Turkish minor. Along with this regional public health complex, one American, the Mattel of the University of California (Los Angeles), and another British, the Great Ormond Stree…

Gene therapy could extend the lives of children with rare immune disorder

An investigational gene therapy has restored immune function in all nine children with severe leukocyte adhesion deficiency-I, a rare immune disorder A new trial, led by UCL, Great Ormond Street Hospital for Children (GOSH), Hospital Infantil Universitario Niño Jesús in Madrid, and UCLA, involved nine children aged five months to nine years with severe leukocyte adhesion deficiency I (LAD-I) and tested the effects of gene therapy on the rare imm…

Solve-RD project helps scientists solve 500 extremely rare disease cases

In a groundbreaking European project, over 500 people living with mysterious medical conditions have finally received a diagnosis. These patients suffered from rare disorders such as neurological diseases, muscle conditions, intellectual disabilities, and inherited gastrointestinal cancers. After years—sometimes even decades—of uncertainty, science has brought them clarity, hope, and, in some cases, the chance of treatment. This success didn’t c…