Advantages and drawbacks of AAV vector-mediated gene transfer

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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

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GeneMedi's AAV vector system

GeneMedi offers AAV(Adeno-Associated Virus) vector system and AAV Vector Systems-Kanamycin Resistance (AAVKan system), the serotype includes:

Common Serotypes	AAV1, AAV2, AAV4, AAV5, AAV6, AAV6.2, AAV6.2FF, AAV8, AAV9, AAV-Rh.10, AAV-DJ, AAV-Retro (Retrograde), AAV-PHP.B, AAV-PHP.eB, AAV-PHP.S, AAV-BR1, AAV-2i8, AAV-SIG, AAV-VEC.
AAV2 Variants	AAV2 variant (Y444F), AAV2 variant (Y272F, Y444F, Y500F, Y730F), AAV2 variant (Y444F, Y730F, Y500F, Y272F, Y704F, Y252F), AAV2 variant (AAV2.7m8).
AAV8 Variants	AAV8-1m, AAV8-2m, AAV8 variant (Y733F, Y447F, Y275).
MyoAAV Series	AAV-MyoAAV1A, AAV-MyoAAV2A, AAV-MyoAAV3A, AAV-MyoAAV4A, AAV-MyoAAV4E.
Other Specialized Variants	AAV-Anc80, AAV-rh74., AAV-LK03

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Knowledge bases

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AAV Knowledge Base CRISPR/Cas9-gRNA system Knowledge Base

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