Module 9 2021

Designs for GT therapeutic strategies

Ex-vivo transduction (strimvelis)

Therapeutic transgene packaged into a (viral) vector

Packaging cell

Expression system

Transduction

WCB

Haematopoietic (Stem) cell origin

In-vivo transduction

…and injected into the patient

Haematopoietic (Stem) cell origin

(Glybera)

Genetically modified cells are expanded in vitro

…and re-infused into the patient

Patient

Donor

Novartis Pharma AG- Business use only- not for further distribution

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