AbbreviationsγRV

γ-Retroviral Vector;

AAV

Adeno-associated viral vector

AAVS1

Adeno-associated virus site 1

ADA

Adenosine deaminase deficiency

ART-SCID

Artemis severe combined immunodeficiency

ATMP

Advanced therapy medicinal product

CGD

Chronic granulomatous disease

CRISPR/Cas

Clustered regularly interspaced short palindromic repeats

EF1α

Elongation factor 1α

ERT

Enzyme replacement therapy

GMP

Good-manufacturing practice

GvHD

Graft versus host disease

HDR

Homology-directed repair

HSCT

Hematopoietic stem cell transplantation

HSPCs

Hematopoietic stem progenitor cells

IDLV

Integrase defective lentiviral vector

IEI

Inborn errors of immunity

IEWP

Inborn errors working party

IPEX

Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome

iPSCs

Induced pluripotent stem cells

ITGB2,

Integrin subunit beta 2

LAD

Leukocyte adhesion deficiency

LTRs

Long-terminal repeats

MMLV

Moloney murine leukemia virus

NADPH

Nicotinamide adenine dinucleotide phosphate oxidase

NHEJ

Non-homologous end joining

PAM

Protospacer adjacent motif

PEG-ADA

Polyethylene glycol-conjugated bovine enzyme

RAG1

Recombination Activating Genes 1

RAG2

Recombination Activating Genes 2

RIC

Reduced intensity conditioning

SCID-X1

X-linked severe combined immunodeficiency

SCID

Severe combined immunodeficiency

ssODN

Single-stranded oligodeoxynucleotide

TALENs

Transcription activator-like effector nucleases

WAS

Wiskott-Aldrich syndrome

WASp

Wiskott-Aldrich syndrome protein

WPRE

Woodchuck post-transcriptional regulatory element

X-HIGM or HIGM1

X-linked hyper-immunoglobulin M (IgM) syndrome

ZFNs

Zinc finger nucleases

Keywords

[Primary immunodeficiency

Inborn errors of immunity

Hematopoietic stem cell transplantation

Gene therapy

Lentiviral vectors

Retroviral vectors

Gene editing

CRISPR/Cas9]