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Miguel Sena-Esteves, PhD

Research Focus - Gene Therapy for Neurological Diseases and Brain Tumors

  • Development of adeno-associated virus (AAV)-based gene therapy for ultra-rare neurological diseases 
  • Translation of gene therapy programs from bench to bedside
  • Directed in vivo evolution of AAV capsid libraries for development of targeted therapies for the central nervous system, retina, and skeletal muscle
  • AAV gene delivery of therapeutic genes to the brain tumor microenvironment to control growth and elicit therapeutic responses
  • Utilization of antisense oligonucleotides and siRNAs for in vivo targeting of genetic networks that drive glioblastoma and diffuse intrinsic pontine glioma growth

Representative Publication

Golebiowski D, van der Bom IMJ, Kwon CS, et al. Direct intracranial injection of AAVrh8 encoding monkey β-N-acetylhexosaminidase causes neurotoxicity in the primate brain. Hum Gene Ther. 2017;28(6):510-522.

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