γ-Retroviral Vector;
AAVAdeno-associated viral vector
AAVS1Adeno-associated virus site 1
ADAAdenosine deaminase deficiency
ART-SCIDArtemis severe combined immunodeficiency
ATMPAdvanced therapy medicinal product
CGDChronic granulomatous disease
CRISPR/CasClustered regularly interspaced short palindromic repeats
EF1αElongation factor 1α
ERTEnzyme replacement therapy
GMPGood-manufacturing practice
GvHDGraft versus host disease
HDRHomology-directed repair
HSCTHematopoietic stem cell transplantation
HSPCsHematopoietic stem progenitor cells
IDLVIntegrase defective lentiviral vector
IEIInborn errors of immunity
IEWPInborn errors working party
IPEXImmune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome
iPSCsInduced pluripotent stem cells
ITGB2,Integrin subunit beta 2
LADLeukocyte adhesion deficiency
LTRsLong-terminal repeats
MMLVMoloney murine leukemia virus
NADPHNicotinamide adenine dinucleotide phosphate oxidase
NHEJNon-homologous end joining
PAMProtospacer adjacent motif
PEG-ADAPolyethylene glycol-conjugated bovine enzyme
RAG1Recombination Activating Genes 1
RAG2Recombination Activating Genes 2
RICReduced intensity conditioning
SCID-X1X-linked severe combined immunodeficiency
SCIDSevere combined immunodeficiency
ssODNSingle-stranded oligodeoxynucleotide
TALENsTranscription activator-like effector nucleases
WASWiskott-Aldrich syndrome
WASpWiskott-Aldrich syndrome protein
WPREWoodchuck post-transcriptional regulatory element
X-HIGM or HIGM1X-linked hyper-immunoglobulin M (IgM) syndrome
ZFNsZinc finger nucleases
Keywords[Primary immunodeficiency
Inborn errors of immunity
Hematopoietic stem cell transplantation
Gene therapy
Lentiviral vectors
Retroviral vectors
Gene editing
CRISPR/Cas9]
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