Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing
Highlights From the ESGCT 28th Annual Congress

Monitoring of Vector Integration Sites in In Vivo Gene Therapy Approaches by Liquid-Biopsy-Integration-Site Sequencing

D Cesana1, A Calabria1, L Rudilosso1, P Gallina1, G Spinozzi1, A Magnani2, M Pouzolles3, F Fumagalli1, V Calbi1, M Witzel4, F D Bushman5, A Cantore1, N Taylor3, V S Zimmermann3, C Klein4, A Fischer2, M Cavazzana2, E Six2, A Aiuti1, L Naldini1, E Montini1

1San Raffaele Telethon Insitute for Gene Therapy (HSR-TIGET)

2Laboratory of Human Lymphohematopoiesis, INSERM, France

3Institut de Génétique Moléculaire de Montpellier, CNRS, France

4Dr. von Hauner Children's Hospital, LMU, Germany

5Department of Microbiology, UPenn, USA

Key Data Points

IS Retrieval From Cell-free DNA Purified From Body Fluids

Cell-free DNA (cfDNA) is released into the blood from healthy, inflamed, or diseased solid organ cells (such as liver or thymus) undergoing apoptosis or necrosis. Genetic alterations in purified cfDNA, including vector–genomic integration sites (IS) from transduced cells, can be detected by liquid biopsy integration site sequencing (LiBIS-seq).

Characterization of Lentivirus (LV) Integration Profile in Hemophilic Dogs

Three adult hemophilic dogs (O21, M57, and O59) received a single injection of LVs expressing different versions of canine coagulation FIX. For each dog, cfDNA was purified from blood serum harvested at 7–8 different time points starting from 30 to 532 d after injection. This graph shows the estimated population size of marked cell clones contributing to the shared ISs over time in dogs M57 and O59.

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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