Viral vectors |
p53 |
Adenovirus |
High |
High |
Induces apoptosis |
Adenovirus is a commonly used vector for gene therapy due to its high transfection efficiency and ability to infect a wide range of cells |
The use of adenovirus for p53 gene therapy is a novel approach that has shown promising results in preclinical studies |
High transfection efficiency results in high gene expression levels and improved therapeutic outcomes |
Adenovirus can induce an immune response, limiting its effectiveness and potential toxicity |
[49, 50] |
Non-viral vectors |
siRNA |
Lipid nanoparticles |
Moderate |
Moderate |
Downregulates target gene expression |
Lipid nanoparticles are a promising non-viral vector for gene therapy due to their biocompatibility and ability to encapsulate and protect nucleic acids |
The use of lipid nanoparticles for siRNA delivery is a relatively new approach that has shown improved transfection efficiency and gene silencing compared to other non-viral vectors |
Lower risk of immune response and toxicity compared to viral vectors |
Lower transfection efficiency and gene expression levels compared to viral vectors |
[49, 50] |
CRISPR-Cas9 |
PD-1 |
Gold nanoparticles |
High |
High |
Enhances T cell activity |
Gold nanoparticles have unique optical and electronic properties that make them promising candidates for gene therapy |
The use of gold nanoparticles for CRISPR-Cas9 delivery is a cutting-edge approach that has shown promising results in preclinical studies |
High transfection efficiency and gene expression levels |
Limited data on long-term safety and potential toxicity |
[117] |
Electroporation |
IL-12 |
Electric pulses |
Moderate |
High |
Induces immune response |
Electroporation is a non-viral method for delivering nucleic acids into cells using brief electric pulses |
The use of electroporation for IL-12 gene therapy is a novel approach that has shown promising results in preclinical studies |
Non-toxic and non-immunogenic |
Requires specialized equipment and expertise |
[251] |
CRISPR-Cas13a |
KRAS |
Liposomes |
High |
Moderate |
Downregulates target gene expression |
CRISPR-Cas13a is a recently discovered RNA-guided ribonuclease system that can be used for RNA editing |
The use of CRISPR-Cas13a for KRAS gene therapy is a cutting-edge approach that has shown promising results in preclinical studies |
Specific and efficient targeting of RNA |
Limited data on long-term safety and potential off-target effects |
[252] |
AAV vectors |
BDNF |
Adeno-associated virus |
High |
High |
Enhances neuronal growth and survival |
Adeno-associated virus (AAV) vectors are a type of viral vector that can be used for gene therapy due to their safety and ability to integrate into the host genome |
The use of AAV vectors for brain-derived neurotrophic factor (BDNF) gene therapy is a promising approach for treating neurodegenerative diseases |
Long-term expression and stability |
Limited packaging capacity and potential immune response |
[253] |
Hybrid viral vectors |
HER2 |
Hybrid viral vectors |
High |
High |
Induces apoptosis |
Hybrid viral vectors combine the advantages of different viral vectors to achieve improved transduction efficiency and specificity |
The use of hybrid viral vectors for HER2 gene therapy is a promising approach for treating HER2-positive breast cancer |
Improved transduction efficiency and specificity compared to single viral vectors |
Potential immune response and toxicity |
[254] |
CRISPR-Cas9 |
LDLR |
Gold nanorods |
Moderate |
Moderate |
Upregulates target gene expression |
Gold nanorods are a type of gold nanoparticle that can be used for gene therapy due to their plasmonic properties |
The use of gold nanorods for CRISPR-Cas9-mediated LDLR gene therapy is a novel approach that has shown promising results in preclinical studies |
Non-toxic and biocompatible |
Limited data on long-term safety and efficacy |
[117] |
Non-viral vectors |
BRCA1 |
Polymeric nanoparticles |
High |
High |
Induces DNA repair |
Polymeric nanoparticles are a type of non-viral vector that can be used for gene therapy due to their versatility and biocompatibility |
The use of polymeric nanoparticles for BRCA1 gene therapy is a promising approach for treating breast cancer |
High transfection efficiency and biocompatibility |
Limited packaging capacity and potential toxicity |
[49, 50] |
In vivo electroporation |
IL-10 |
In vivo electroporation |
High |
High |
Suppresses inflammation |
In vivo electroporation is a non-viral method for delivering nucleic acids into cells in vivo using electric pulses |
The use of in vivo electroporation for IL-10 gene therapy is a promising approach for treating inflammatory diseases |
Non-toxic and non-immunogenic |
Limited data on long-term safety and potential toxicity |
[255] |
CRISPR-Cas12a |
EGFR |
Lipid nanoparticles |
High |
Moderate |
Downregulates target gene expression |
CRISPR-Cas12a is a recently discovered RNA-guided endonuclease system that can be used for gene editing |
The use of CRISPR-Cas12a for EGFR gene therapy is a novel approach that has shown promising results in preclinical studies |
Specific and efficient targeting of DNA |
Limited data on long-term safety and potential off-target effects |
[256] |
mRNA-based vaccines |
SARS-CoV-2 spike protein |
Lipid nanoparticles |
High |
High |
Induces immune response |
mRNA-based vaccines are a novel approach to gene therapy that use messenger RNA (mRNA) to encode a protein of interest and induce an immune response |
The use of mRNA-based vaccines for the SARS-CoV-2 spike protein is a cutting-edge approach for preventing COVID-19 |
Rapid and scalable production |
Potential immune response and toxicity |
[257] |
CRISPR-Cas9 |
HPRT |
Zinc-finger nucleases |
Moderate |
Moderate |
Gene correction |
Zinc-finger nucleases (ZFNs) are a type of engineered DNA-cutting enzyme that can be used for gene editing |
The use of ZFNs for HPRT gene therapy is a promising approach for treating genetic disorders |
Specific and efficient targeting of DNA |
Limited data on long-term safety and potential off-target effects |
[117, 258] |
Viral vectors |
CFTR |
Lentivirus |
High |
High |
Induces gene expression |
Lentivirus is a type of retrovirus that can be used as a vector for gene therapy |
The use of lentivirus for CFTR gene therapy is a promising approach for treating cystic fibrosis |
High transduction efficiency and stable gene expression |
Potential immune response and toxicity |
[259] |
Non-viral vectors |
VEGF |
Dendrimers |
Moderate |
Moderate |
Enhances angiogenesis |
Dendrimers are a type of branched polymer that can be used as a non-viral vector for gene therapy |
The use of dendrimers for VEGF gene therapy is a novel approach that has shown promising results in preclinical studies |
Highly customizable and biocompatible |
Limited transfection efficiency and potential toxicity |
[260] |
CRISPR-Cas9 |
DMD |
AAV vectors |
High |
High |
Gene correction |
Adeno-associated virus (AAV) vectors are a type of viral vector that can be used for gene therapy |
The use of AAV vectors for DMD gene therapy is a promising approach for treating Duchenne muscular dystrophy |
Long-term gene expression and safety |
Limited packaging capacity and potential immune response |
[117] |
mRNA-based therapies |
OCT4 |
mRNA electroporation |
High |
High |
Induces cell reprogramming |
mRNA-based therapies are a novel approach to gene therapy that use messenger RNA (mRNA) to encode a protein of interest |
The use of mRNA electroporation for OCT4 gene therapy is a cutting-edge approach for inducing cell reprogramming |
Rapid and customizable production |
Potential immune response and toxicity |
[261] |
Non-viral vectors |
MDR1 |
Nanoparticles |
Moderate |
Moderate |
Downregulates target gene expression |
Nanoparticles are a type of non-viral vector that can be used for gene therapy due to their size and biocompatibility |
The use of nanoparticles for MDR1 gene therapy is a promising approach for overcoming multidrug resistance in cancer |
Non-toxic and biocompatible |
Limited transfection efficiency and gene expression levels |
[49, 50] |
CRISPR-Cas9 |
HBB |
Lipid nanoparticles |
High |
High |
Gene correction |
Lipid nanoparticles are a type of non-viral vector that can be used for gene therapy due to their biocompatibility and ease of production |
The use of lipid nanoparticles for HBB gene therapy is a promising approach for treating sickle cell disease |
Non-immunogenic and scalable production |
Limited transfection efficiency and gene expression levels |
[117] |
Gene silencing |
BCL-2 |
Aptamers |
High |
Moderate |
Downregulates target gene expression |
Aptamers are a type of synthetic nucleic acid that can be used as a gene silencing agent |
The use of aptamers for BCL-2 gene therapy is a novel approach that has shown promising results in preclinical studies |
Specific and efficient targeting of RNA |
Limited in vivo stability and potential immunogenicity |
[262] |
Non-viral vectors |
FGF2 |
Cationic liposomes |
Moderate |
Moderate |
Enhances angiogenesis |
Cationic liposomes are a type of non-viral vector that can be used for gene therapy due to their ability to interact with cell membranes |
The use of cationic liposomes for FGF2 gene therapy is a promising approach for promoting tissue regeneration |
Low toxicity and customizable |
Limited transfection efficiency and stability |
[49, 50, 263] |
CRISPR-Cas9 |
CFTR |
CRISPR-Cas9 ribonucleoprotein |
High |
High |
Gene correction |
CRISPR-Cas9 ribonucleoprotein is a recently developed gene editing technology that uses RNA-guided endonucleases |
The use of CRISPR-Cas9 ribonucleoprotein for CFTR gene therapy is a promising approach for treating cystic fibrosis |
High specificity and efficiency |
Potential off-target effects and immune response |
[117, 264] |
RNA interference |
KRAS |
Gold nanoparticles |
Moderate |
Moderate |
Downregulates target gene expression |
Gold nanoparticles are a type of nanoparticle that can be used for gene therapy due to their unique optical and electronic properties |
The use of gold nanoparticles for KRAS gene therapy is a novel approach that has shown promising results in preclinical studies |
High biocompatibility and stability |
Limited transfection efficiency and potential toxicity |
[194, 195] |
Viral vectors |
APOE |
Adenovirus |
High |
High |
Upregulates target gene expression |
Adenovirus is a type of viral vector that can be used for gene therapy due to its high transduction efficiency |
The use of adenovirus for APOE gene therapy is a promising approach for treating Alzheimer's disease |
High transduction efficiency and long-term gene expression |
Potential immune response and toxicity |
[265] |
RNA interference |
TP53 |
Nanoparticles |
High |
High |
Downregulates target gene expression |
Nanoparticles are a type of non-viral vector that can be used for gene therapy due to their size and biocompatibility |
The use of nanoparticles for TP53 gene therapy is a promising approach for treating various cancers |
Non-toxic and biocompatible |
Limited transfection efficiency and gene expression levels |
[193] |
Gene editing |
F9 |
CRISPR-Cas9 |
High |
High |
Gene correction |
CRISPR-Cas9 is a gene editing technology that uses RNA-guided endonucleases to modify DNA |
The use of CRISPR-Cas9 for F9 gene therapy is a promising approach for treating hemophilia B |
High specificity and efficiency |
Potential off-target effects and immune response |
[266] |
mRNA-based therapies |
FLT3L |
mRNA electroporation |
High |
High |
Induces immune response |
mRNA-based therapies are a novel approach to gene therapy that use messenger RNA (mRNA) to encode a protein of interest |
The use of mRNA electroporation for FLT3L gene therapy is a cutting-edge approach for enhancing immune response |
Rapid and customizable production |
Potential immune response and toxicity |
[194, 195] |