ADA	Adenosine Deaminase Deficiency
ATMP	Advanced Therapy Medicinal Product
BM	Bone Marrow
CAR	Chimeric Antigen Receptor
CAT	Committee for Advanced Therapies
CCMO	Centrale Commissie Mensgebonden Onderzoek
CGD	Chronic Granulomatous Disease
CHMP	Committee for Human Medicinal Products
CTA	Clinical Trial Application
ddPCR	Digital Droplet PCR
DL1	Delta-Ligand 1
EC	European Commission
EMA	European Medicines Agency
EU	European Union
FDA	U.S. Food and Drug Administration
FTOC	Fetal Thymus Organ Culture
GMO	Genetically Modified Organism
GMP	Good Manufacturing Practice
GvHD	Graft versus Host Disease
HLA	Human Leukocyte Antigen
HSC	Hematopoietic Stem Cell
HSPC	Hematopoietic Stem Progenitor cell
IB	Investigator Brochure
IMPD	Investigational Medicinal Product Dossier
iPSCS	Induced Pluripotent Stem Cell
IVIM	In Vitro Immortalization Assay
LAM-PCR	Linear Amplification Mediated PCR
LSK	Lin−Sca+cKit+ cells
LTR	Long Terminal Repeat
LUMC	Leiden University Medical Center
LV	Lentiviral vector
MA	Marketing Authorization
mPB	Mobilized Peripheral Blood
NSG	NOD Scid Gamma
nrLAM-PCR	Non-restrictive LAM-PCR
PCR	Polymerase Chain Reaction
PRIME	Priority Medicines
RAG1/2	Recombinase-Activating Gene 1/2
RV	Retroviral Vector
SAGA	Surrogate Assay for Genotoxicity Assessment
SCID	Severe Combined Immunodeficiency
SIN	Self-Inactivating
TE	Transduction enhancer
USA	United States of America
VCN	Vector Copy Number
VHP	Voluntary Harmonized Procedure
WAS	Wiskott–Aldrich Syndrome
WPRE	Woodchuck hepatitis virus Post-transcriptional Regulatory Element