| Gene therapy (Transient expression)
|
U937 cells |
– |
eGFP
|
Nordin et al. (2016) and Nordin, Karim & Wahid (2014)
|
|
Retinal Pigment epithelium in mice |
Rpe65 deficiency |
eGFP and Rpe65
|
Yanez-Munoz et al. (2006)
|
| Haemophilia B Mice |
Factor IX (FIX) deficiency of haemophiliac V |
Human FIX cDNA
|
Suwanmanee et al. (2014)
|
| Cell Reprogramming |
HepG2 cells, fibroblast |
Induce pluripotent states that resembles human embryonic stem (hES) cells |
Yamanaka’s factors, SV40 Tag |
Mali et al. (2008)
|
|
U937 cells |
Induce pluripotency |
Yamanaka’s factors |
Nordin (2011)
|
|
Bone marrow MSC |
Induce pluripotency via Yamanaka’s factors but flanked by loxP sites between LTRs |
Cre recombinase |
Papapetrou & Sadelain (2011)
|
| Hepatic progenitor cells |
Sort and select human hepatic progenitor cells |
GFP driven by liver-specific apolipoprotein A-II (APOA-II) promoter |
Yang et al. (2013)
|
| Cell death |
293T cells |
Selective elimination of tumour cells |
Thymidine kinase (TK) suicide gene |
Vargas Jr, Klotman & Cara (2008)
|
| Gene editing (Flp recombinase)
|
HEK-293 and 293T |
Integration of circular DNA |
Flp recombinase |
Moldt et al. (2008)
|
| Gene editing (I-SceI nuclease) |
U2-OS, HEK293, HT-1080, and HEK293T cells |
Defective enhanced green fluorescent protein (EGFP) gene |
I-SceI nuclease and eGFP |
Cornu & Cathomen (2007)
|
| Gene editing (Sleeping Beauty (SB) transposase) |
HeLa cells |
Abrogate chromosomal |
Sleeping Beauty (SB) transposase |
Vink et al. (2009)
|
| Gene editing (Piggyback (PB) DNA transposase) |
HEK293 and HeLa cells |
Puromycin screening |
Puromycin resistant gene |
Skipper et al. (2018)
|
| Gene editing (Zinc Finger Nuclease)
|
Epstein-Barr virus–transformed B lymphocytes (lymphoblastoid cells), cord blood CD34+ hematopoietic progenitor cells, human ES cell lines (HUES-3 and HUES-1) |
Severe combined immunodeficiencies (SCID) |
IL2RG with a silent point mutation, GFP or PuroR
|
Lombardo et al. (2007)
|
|
Human repopulating hematopoietic CD341 stem cells |
X-linked severe combined immunodeficiency (SCID-X1) |
AAVS1, IL2RG
|
Genovese et al. (2014)
|
| CD34+ hematopoietic cells |
Fanconi anaemia (FA) |
FANCA expression cassette |
Diez et al. (2017)
|
| Gene editing (Transcription activator-like effector nuclease (TALEN)) |
Patient derived T-lymphocytes |
X-linked hyper-immunoglobulin M (hyper-IgM) syndrome (XHIM) |
CD40LG
|
Kuo et al. (2018)
|
| Primary fibroblast |
Recessive dystrophic epidermolysis bullosa (RDEB) |
COL7A1
|
Osborn et al. (2013)
|
| Gene editing (Clustered regularly interspaced short palindrome repeats-Cas9 (CRISPR-Cas9))
|
Defective keratinocytes |
Junctional epidermolysis bullosa (JEB) |
LAMB3
|
Benati et al. (2018)
|
|
B-lymphocytes |
Eradicating tumour cells |
a-PD1
|
Luo et al. (2020)
|
|
HEK293 |
Off-target nuclease activity sites |
Puromycin-resistance gene |
Wang et al. (2015)
|
|
HEK293T cells and post-mitotic brain neurons |
Off-target nuclease activity sites |
All-in-one (sgRNA and Cas9) vector cassette with SP1 binding site |
Ortinski et al. (2017)
|
|
Colorectal cancer stem cells (CRCSC) |
in vivo silencing, investigating the stem-like tumour exosomes secretion |
RAB27B
|
Cheng et al. (2019)
|
|
CRC patient-derived xenografts (PDXs) |
Gene silencing |
ARID3B
|
Liao et al. (2020)
|
|
Induced pluripotent stem cell (iPSC) derived from patients with alpha-synuclein gene (SNCA) locus triplication |
Disease model of Parkinson’s |
CRISPR-dCas9-DNA methylation system |
Tagliafierro, Zamora & Chiba-Falek (2019)
|
| Sickle cells |
Correction of mutation |
βs-globin
|
Uchida et al. (2021)
|
| Immunization or vaccination |
BALB/c mice |
HIV-1 infection |
Codon optimized HIV-1JR-FL gp120 |
Negri et al. (2007)
|
|
Mice |
West Nile Virus (WNV) infection |
Gene encoding secreted form of envelope of virulent strain of West Nile Virus (WNV) |
Coutant et al. (2008)
|
|
Mice |
HCV infection |
HCV E1/E2 pseudotyped IDLV bearing HCV NS3 gene |
Deng et al. (2013)
|
|
CB6F1 mice |
Influenza virus |
IDLV pseudotyped with virus hemagglutinin (HA) and nucleoprotein (NP) |
Gallinaro et al. (2018)
|
|
Mice |
Influenza A virus (IAV) |
Genes encoding for VN04-2 monoclonal antibodies |
Michelini et al. (2020)
|
|
C57BL/6J mice |
SARS-COV-2 virus |
LV-derived virus simulating particles (VSPs) decorated with SARS-COV-2 spike protein and carrying spike encoding mRNA |
Yin et al. (2020)
|
|
Mice and golden hamsters |
SARS-COV-2 virus |
Spike glycoprotein gene |
Ku et al. (2021c)
|
|
Mice |
Lymphoma and melanoma |
Ovalbumin (OVA), TRP2
|
Morante et al. (2021)
|
| C57BL/6 mice |
Anti-tumour immunity |
OVA
|
Cousin et al. (2019)
|
