Advantages and drawbacks of AAV vector-mediated gene transfer

pdf download AAV protocol Download

Adeno Associated Virus (AAV)

a) Advantages of AAV-mediated gene transfer
AAV has been developed into a very attractive candidate for creating viral vectors for gene therapy and the creation of isogenic human disease models due to various advantages. 1) Superior biosafety rating. The wild type AAV has not currently been known to cause disease in vivo, and further security of recombinant AAV gene delivery in vivo is ensured after removal of most AAV genome elements. 2) Low immunogenicity. AAV causes a very mild immune response in vivo, lending further support to its apparent lack of pathogenicity during gene delivery. 3) Broad range of infectivity. AAV can infect both dividing and quiescent cells in vivo, allowing gene delivery to a highly diverse range of cell types. 4) Stable expression. Long term gene delivery in vivo can be mediated by AAV. b) Drawbacks of AAV-mediated gene transfer
Although adenovirus benefits a great deal of disease therapies, it does present some drawbacks.
1) The major drawback is its limited cloning capacity (less than 4.7kb) of the vector, which restricts its use in gene delivery of large genes.(Table 3) [32]. 2) Generation of neutralizing antibodies against AAV in the Non-Human Primates (NHP) and human, may attenuate the cure effect of AAV-mediated gene therapy [33].

Table 2. Comparison between Retrovirus, Lentivirus, Adenovirus and Adeno-associated virus (AAV) vectors.
Comparison Retrovirus Lentivirus Adenovirus AAV
Genome ss RNA ss RNA ds DNA ss DNA
Integration Yes Yes No No
Packaging Capacity 3kb 4kb 5.5kb 2kb
Time to peak expression 72h 72h 36h-72h Cell: 7 days;
Animals: 2 weeks
Sustainable time About 3 weeks Stable expression Transient expression > 6 months
Cell Type Most Dividing Most Dividing/Non-Dividing Cells Most Dividing/Non-Dividing Cells Most Dividing/Non-Dividing Cells
Titer 10^7 TU/ml 10^8 TU/ml 10^11 PFU/ml 10^12 vg/ml
Animal experiment Suitable Low efficiency Lowest efficiency Most suitable
Immune Response High Medium Medium Ultra-low

GeneMedi's AAV Products with different serotypes


AAV(Adeno-Associated Virus) vector system


AAV1 vector system

AAV2 vector system

AAV2 variant(Y444F) vector system

AAV2 variant (Y272F,Y444F,Y500F,Y730F) vector system

AAV2 variant(Y444F,Y730F,Y500F,Y272F,Y704F,Y252F) vector system

AAV2 variant(AAV2.7m8) vector system

AAV5 vector system

AAV6 vector system

AAV8 vector system

AAV8-1m vector system

AAV8-2m vector system

AAV8-3m vector system



AAV9 vector system

AAV-Rh10 vector system

AAV-DJ vector system

AAV-Dj8 vector system

AAV2-Retro (Retrograde) vector system

AAV-PHP.B vector system

AAV-PHP.eB vector system

AAV-PHP.S vector system

AAV-BR1 vector system

AAV-2i8 vector system

AAV-SIG vector system

AAV-VEC vector system



AAV Rep-Cap plasmids (serotypes-specific AAV RC plasmids)


AAV1 Rep-Cap Plasmid

AAV2 Rep-Cap Plasmid

AAV2 variant (Y444F) Rep-Cap plasmid

AAV2 variant (Y272F, Y444F, Y500F, Y730F) Rep-Cap plasmid

AAV2 variant (Y444F, Y730F, Y500F, Y272F, Y704F, Y252F) Rep-Cap plasmid

AAV2 variant(AAV2.7m8) Rep-Cap plasmid

AAV5 Rep-Cap Plasmid

AAV6 Rep-Cap Plasmid

AAV8 Rep-Cap Plasmid

AAV8-1m Rep-Cap plasmid

AAV8-2m Rep-Cap plasmid

AAV8 variant (Y733F, Y447F, Y275) Rep-Cap plasmid



AAV9 Rep-Cap Plasmid

AAV-Rh.10 Rep-Cap Plasmid

AAV-DJ Rep-Cap Plasmid

AAV-DJ/8 Rep-Cap Plasmid

AAV-Retro (Retrograde) Rep-Cap plasmid

AAV-PHP.B Rep-Cap plasmid

AAV-PHP.eB Rep-Cap plasmid

AAV-PHP.S Rep-Cap plasmid

AAV-BR1 Rep-Cap plasmid

AAV-2i8 Rep-Cap plasmid

AAV-SIG Rep-Cap plasmid

AAV-VEC Rep-Cap plasmid



GM TransExcellent Plasmid DNA Rapid Preparation Service

GeneMedi offers a plasmid production&lification service called GM TransExcellent Plasmid DNA Rapid Preparation Service for DNA vector preparation based on the G-NEXT™ platform, which is a fast (1 week), high-quality, high-efficiency, and cost-effective plasmid production&lification platform and is good for your proteins, antibodies, and viral vector production. More Details About the Service >>

View Adeno-associated virus (AAV vector) Knowledge Base>>

Other knowledge bases

AAV Knowledge Basepdf download
CRISPR/Cas9-gRNA system Knowledge Base
Adenovirus Knowledge Basepdf download
Transfection Knowledge Base
Lentivirus Knowledge Basepdf download
Advanced cell therapy


View Adeno-associated virus (AAV vector) Knowledge Base>>

Other knowledge bases

AAV Knowledge Basepdf download
CRISPR/Cas9-gRNA system Knowledge Base
Adenovirus Knowledge Basepdf download
Transfection Knowledge Base
Lentivirus Knowledge Basepdf download
Advanced cell therapy




GENEMEDI
Email: [email protected]   [email protected]
Telephone: +86-21-50478399   Fax: 86-21-50478399
Privacy Policy
<