GENE THERAPIES

Sponsored By 

Concurrent Speaker(s)

• 

  Benedikt Berninger, PhD (he/him/his)

  Centre for Developmental Neurobiology
  King's College London, UK
• 

  Dimitri Kullmann, MD, PhD

  UCL Queen Square Institute of Neurology
  University College London

Presenting Author(s)

• 

  Thomas Berger, PhD

  Research & Development
  Catalent Cell & Gene Therapy, Germany
• 

  Carine Bourdais

  IRMB
  Université de Montpellier, INSERM, CHU Montpellier, France
• 

  Michela Milani, PhD (she/her/hers)

  San Raffaele Telethon Institute for Gene Therapy
  IRCCS San Raffaele Hospital, Italy
• AR

  Alessandra Ricca, PhD

  San Raffaele Telethon Institute for Gene Therapy
  San Raffaele hospital, Italy

This session will focus on emerging new areas of gene therapy, in the clinic or advancing towards the clinic

Presentations:

• ENGINEERING NEURAL CELL FATES BY IN VIVO LINEAGE REPROGRAMMING
  

  Concurrent Speaker: Benedikt Berninger, PhD (he/him/his) – King's College London
• LENTIVIRAL VECTOR MEDIATED IN VIVO GENE TRANSFER INTO HEMATOPOIETIC STEM AND PROGENITOR CELLS FOR THE TREATMENT OF FANCONI ANEMIA
  

  Presenting Author: Michela Milani, PhD (she/her/hers) – IRCCS San Raffaele Hospital
• TESTING LENTIVIRAL VECTORS ENCODING FOR CHIMERIC HUMAN GALC ENZYMES TO REFINE HEMATOPOIETIC STEM CELL GENE THERAPY FOR GLOBOID CELL LEUKODYSTROPHY.
  

  Presenting Author: Alessandra Ricca, PhD – San Raffaele hospital
• COMBINED CELLULAR AND GENE THERAPY TO TREAT PRIMARY CILIARY DYSKINESIA.
  

  Presenting Author: Carine Bourdais – Université de Montpellier, INSERM, CHU Montpellier
• OPTIMIZATION OF A GMP-COMPATIBLE GENE EDITING METHODOLOGY TO GENERATE HUMAN INDUCED PLURIPOTENT STEM CELLS WITH ENHANCED FEATURES FOR THERAPEUTIC APPLICATIONS
  

  Presenting Author: Thomas Berger, PhD – Catalent Cell & Gene Therapy
• GENE THERAPY FOR FOCAL REFRACTORY EPILEPSY
  

  Concurrent Speaker: Dimitri Kullmann, MD, PhD – University College London