Cell & Gene Therapy - First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicine
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Discover Bioprinted Liver Models for Drug DevelopmentDiscover Bioprinted Liver Models for Drug Development

Animal models often fail to predict human drug toxicity. Discover how advanced bioprinted liver models, combining stem cells, biofabrication, engineered hydrogels, and AI, are creating more physiologically relevant platforms for preclinical testing. Explore emerging approaches that could improve toxicity prediction and accelerate the development of safer, more effective therapies.

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   First-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicineFirst-in-the-world gene therapy delivers missing gene directly to infant’s brain, marking historic milestone in precision medicine
 
An eight-month-old baby from Israel has become the first human ever to receive an experimental gene therapy designed to replace a missing gene responsible for a devastating neurological disease that until now offered no hope of survival.
 
   New stem cell therapy offers hope for chronic spinal injuryNew stem cell therapy offers hope for chronic spinal injury
 
New research presented today at the International Society for Stem Cell Research (ISSCR) 2026 Annual Meeting highlights a significant step toward developing a stem cell-based treatment for chronic spinal cord injury (SCI), a condition for which no effective restorative therapy currently exists.
 
 Salt-loaded nanoparticles help deliver fragile gene therapies into cells
 
Salt-loaded nanoparticles help deliver fragile gene therapies into cellsResearchers at the University of Houston's College of Pharmacy have discovered an unexpected simple strategy to improve the performance of mRNA vaccines and gene therapeutics: adding salt.
 
 
 Pioneering gene therapy for rare immune disorder shows promise in early pre-clinical studies
 
Pioneering gene therapy for rare immune disorder shows promise in early pre-clinical studiesThe development of a pioneering gene therapy to treat a devastating, rare immune disorder led by UCL scientists is set to move into its next phase following promising early pre-clinical studies.
 
 
 Gene therapy shows promise against deadly childhood liver disease
 
Gene therapy shows promise against deadly childhood liver diseaseA new gene therapy has been used to successfully treat a deadly childhood liver disease using mice that model this disease by researchers at UCL and Great Ormond Street Hospital.
 
 
 Advanced imaging technology unlocks exosome potential for disease diagnosis
 
Advanced imaging technology unlocks exosome potential for disease diagnosisTiny particles released by most human cells may be promising targets for diagnosis and treatment of an array of diseases from cancer to Alzheimer's, but scientists have struggled to see them and study them in detail.
 
 
 New gene therapy strategy uses the brain's own glymphatic transport system for drug delivery
 
New gene therapy strategy uses the brain's own glymphatic transport system for drug deliveryA new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.
 
 

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