Gene therapy for lysosomal storage disorders: ongoing studies and clinical development

HIGHLIGHTS

• who: Giulia Massaro and colleagues from the UCL School of Pharmacy, University College London, London WC N AX, UK have published the article: Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development, in the Journal: Biomolecules 2021, 611 of /2021/
• what: The general approach of HSC-GT proceeds as follows: autologous HSCs are collected from the patient, collected HSCs are genetically corrected by transduction with a viral_vector harbouring the therapeutic gene and_(3) the transduced HSCs are transplanted back into the patient following transplant conditioning treatment similar to that required for HSCT. This . . .
  

   

  

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