Often, gene therapy reviews concentrate upon specific therapeutic modalities – particularly either viral vector-mediated or a non-viral approach. Here, we draw together a comprehensive array of knowle...

African social spiders’ sinister game of ‘grandmother’s footsteps’ could be breakout moment in BBC’s Parenthood

Our findings indicate that young children with AIPL1-related retinal dystrophy benefited substantially from subretinal administration of rAAV8.hRKp.AIPL1, with improved visual acuity and functional vi...

In type 1 spinal muscular atrophy, pathological changes begin during prenatal development, which means that a complete cure is unlikely even with early pos

The war on science is turning deadly

Preterm birth (PTB; delivery <37 weeks), the main cause of neonatal death worldwide, can lead to adverse neurodevelopmental outcomes, as well as lung and gut pathology. PTB can be associated with asce...

There are currently at least 70 characterised lysosomal storage diseases (LSD) resultant from inherited single-gene defects. Of these, at least 30 present with central nervous system (CNS) neurodegen...

Next generation testing, artificial intelligence, advanced mass spectrometry and machine learning is about to be used for diagnostics in the U.K. thanks to a new partnership between UCLH and Guilford ...

The UCL Great Ormond Street Institute of Child Health and the UCL Faculty of Population Health Sciences are excited to announce the launch of a new MSc programme in Cell, Gene, and Novel Therapies.