uniQure (QURE) publishes a study in Gene Therapy

• QURE announces the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure's AAV5 vector in a large animal model 

 

uniQure announces the online publication in Gene Therapy of data demonstrating widespread transduction in the CNS following direct injection of uniQure's AAV5 vector in a large animal model 
The method of injection used was found to result in very controlled and accurate administration with no adverse events observed in the non-human primates. Varying doses of AAV5 achieved predictable transduction of connected areas of the brain. The data demonstrate that AAV5 is a very effective vector for the central nervous system and has potential for the treatment of a wide range of neurological pathologies. These data will help guide the clinical development of uniQure's gene therapy product candidate AMT-130, which consists of an AAV5 vector carrying an artificial micro-RNA that silences the huntingtin gene for the treatment of Huntington's disease.