AAV

Adeno-Associated Virus serotype 8 (AAV8) was isolated from rhesus monkey tissue, and the AAV8 rep and cap nucleotide sequences have 88% homology with AAV7 and 82% with AAV2. AAV8 exhibits greater transduction efficiency in the liver than other AAV serotypes. AAV8 and 9 have recently been used to correct disease-causing mutations and improve muscle function in mouse models of Duchenne muscular dystrophy. These AAV particles constitutively express the firefly (Photinus pyralis) luciferase and mCherry genes connected via a T2A linker, under the control of a CMV promoter. The T2A self-cleaving peptide (derived from Thosea asigna virus 2A) leads to the efficient cleavage of the transcript and expression of luciferase and mCherry as two separate proteins.

Adeno-Associated Virus serotype 9 (AAV9) is one of the most promising serotypes for gene therapy applications. AAV9 transduces a wide range of tissue types, including cardiac and skeletal muscle, liver, pancreas, and eye tissue. AAV8 and AAV9 have recently been used to correct disease-causing mutations and improve muscle function in mouse models of Duchenne muscular dystrophy. AAV9 has significantly lower seroprevalence in the human population than other AAV serotypes, making AAV9 a desirable candidate for therapeutic applications. These AAV particles constitutively express the firefly (Photinus pyralis) luciferase and mCherry genes connected via a T2A linker, under the control of a CMV promoter. The T2A self-cleaving peptide (derived from Thosea asigna virus 2A) leads to the efficient cleavage of the transcript and expression of luciferase and mCherry as two separate proteins.

Adeno-Associated Virus Serotype 1 (AAV1) exhibits high homology with other AAV serotypes. AAV1 efficiently transduces muscle tissue, as determined by a region of the capsid protein VP1 (amino acids 350 to 430) which functions as a major determinant of tissue tropism. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional inactivation of the gene. SaCas9 (Staphylococcus aureus CRISPR-associated protein 9) has demonstrated high cutting efficiency in mammalian cells, and its smaller size makes it ideal for packaging into AAV. SaCas9 recognizes a longer protospacer adjacent motif (PAM) site,

Adeno-Associated Virus serotype 2 (AAV2) is the best characterized AAV serotype. Nearly all recombinant AAV serotypes utilize the AAV2 inverted terminal repeats (ITRs). AAV2 requires the expression of Heparan Sulfate Proteoglycan (HSPG) on the surface of host for cells for binding and internalization. Of nearly all the discovered AAV serotypes, AAV2 has the best transduction efficiency in cell culture and is the best tool for in vitro studies. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional inactivation of the gene. SaCas9 (Staphylococcus aureus CRISPR-associated protein 9) has dem

Adeno-Associated Virus Serotype 3 (AAV3) shares 82% sequence homology with AAV2, and like AAV2, requires the Heparan Sulfate Proteoglycan (HSPG) receptor for cell attachment. AAV3 vectors transduce human liver cancer cells extremely efficiently because AAV3 utilizes the human Hepatocyte Growth Factor Receptor (hHGFR) as a co-receptor for viral entry, which is highly expressed in these cells. Both the extracellular and intracellular kinase domains of hHGFR are required for AAV3-mediated transgene expression. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional inactivation of the gene. S

Adeno-Associated Virus Serotype 5 (AAV5) differs from other parvovirus serotypes according to serological and DNA hybridization data, and AAV5 also uses different inverted terminal repeats (ITRs) compared to other AAV serotypes. AAV5 is the most efficient vector for transducing sensory neurons and is good at mediating gene transfer into human and murine airway epithelia. In addition, AAV5 vectors show a higher tropism for both mouse and human dendritic cells than AAV1, AAV2, AAV7, or AAV8. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional inactivation of the gene. SaCas9 (Staphylococ

Adeno-Associated Virus serotype 6 (AAV6) appears to be related to AAV1 by sequence analysis and shows the best transduction efficiency in pancreatic beta-cells compared to other AAV serotypes. AAV6 vectors are also particularly effective in the transduction of human prostate, breast, and liver cancer cells. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional inactivation of the gene. SaCas9 (Staphylococcus aureus CRISPR-associated protein 9) has demonstrated high cutting efficiency in mammalian cells, and its smaller size makes it ideal for packaging into AAV. SaCas9 recognizes a longe

Adeno-Associated Virus serotype 8 (AAV8) was isolated from rhesus monkey tissue, and the AAV8 rep and cap nucleotide sequences have 88% homology with AAV7 and 82% with AAV2. AAV8 exhibits greater transduction efficiency in the liver than other AAV serotypes. AAV8 and 9 have recently been used to correct disease-causing mutations and improve muscle function in mouse models of Duchenne muscular dystrophy. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional inactivation of the gene. SaCas9 (Staphylococcus aureus CRISPR-associated protein 9) has demonstrated high cutting efficiency in mamm

Adeno-Associated Virus serotype 9 (AAV9) is one of the most promising serotypes for gene therapy applications. AAV9 transduces a wide range of tissue types, including cardiac and skeletal muscle, and liver, pancreas, and eye tissue. AAV8 and AAV9 have recently been used to correct disease-causing mutations and improve muscle function in mouse models of Duchenne muscular dystrophy. AAV9 has significantly lower seroprevalence in the human population than other AAV serotypes, making AAV9 a desirable candidate for therapeutic applications. Cas9 is an endonuclease enzyme that introduces a double stranded break into the DNA when recruited to a specific DNA sequence by the sgRNA (single guide RNA). This double stranded break is repaired in mammalian cells either through Non-Homologous End Joining or Homologous Recombination. Non-Homologous End Joining often results in the deletion or insertion of several base pairs at the cut site, which, when resulting in a frameshift, causes the functional