How many AAV vectors are there?

How many AAV vectors are there?

Eleven serotypes of AAV have thus far been identified, with the best characterized and most commonly used being AAV2. These serotypes differ in their tropism, or the types of cells they infect, making AAV a very useful system for preferentially transducing specific cell types.

What viral vector is used for hemophilia?

The most advanced clinical trials for gene therapy in haemophilia are using a particular type of carrier, called a vector, to deliver the gene therapy via the bloodstream to liver cells in the body. One important type is known as the AAV (adeno-associated virus) vector.

Is there gene therapy for hemophilia?

A gene therapy treatment restored blood clotting and transformed the disease from severe to mild. Hemophilia is a rare bleeding disorder in which blood doesn’t clot normally. Hemophilia B is caused by mutations in the gene for coagulation factor IX, a protein that helps blood to clot.

Why is AAV used for hemophilia?

To date, the use of AAV in gene therapy for the treatment of hemophilia has been very promising. AAV has irreplaceable advantages compared with other therapeutic methods, including recombinant proteins, neutralizing antibodies, and other viruses, as mentioned above.

How big is the F8 gene?

186 kb
The F8 gene encodes coagulation factor VIII (FVIII). It contains 26 exons, spanning over 186 kb of DNA in the most distal band of the long arm of the X-chromosome (Xq28) [2].

Can Crispr treat hemophilia?

Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be cured by gene therapy. A promising strategy is CRISPR-Cas9-mediated precise insertion of F8 in hepatocytes at highly expressed gene loci, such as albumin (Alb).

What is the F8 gene?

The F8 gene provides instructions for making a protein called coagulation factor VIII. Coagulation factors are a group of related proteins that are essential for the formation of blood clots. After an injury, clots protect the body by sealing off damaged blood vessels and preventing further blood loss.

What is Factor 9 in the blood?

Factor IX is a protein produced naturally in the body. It helps the blood form clots to stop bleeding. Injections of factor IX are used to treat hemophilia B, which is sometimes called Christmas disease. This is a condition in which the body does not make enough factor IX.

What is AAV9 vector?

Adeno-associated virus serotype 9 (AAV9) vector has engendered considerable interest in its therapeutic development for treating neurological disorders (Dayton et al., 2012), in part because it has been heralded as capable of traversing the blood brain barrier to target the central nervous system (CNS), where it has …

What is factor 4 in blood coagulation?

Platelet factor-4 (PF4/CXCL4) is an orphan chemokine released in large quantities in the vicinity of growing blood clots. Coagulation of plasma supplemented with a matching amount of PF4 results in a translucent jelly-like clot.

Why is Christmas a disease?

Hemophilia B, also called factor IX (FIX) deficiency or Christmas disease, is a genetic disorder caused by missing or defective factor IX, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a change in a gene.