University of Example: Hemophilia A Gene Therapy Project Poster

This project focuses on gene therapy for Hemophilia A, a genetic bleeding disorder caused by a deficiency in the factor VIII (FVIII) gene. The assignment explores the use of adeno-associated virus (AAV) as a vector to deliver a functional FVIII gene. The project highlights the process of gene therapy, where a mutated gene is replaced with a functional copy, and the advantages of using AAV, such as its ability to insert genetic material. It also discusses the clinical trials using different dosage groups and the positive outcomes, including reduced bleeding and the need for prophylactic treatments. The project also acknowledges the limitations of AAV, such as its DNA carrying capacity, and references relevant literature on bleeding disorders and immunotherapy. The project is aimed at providing a comprehensive understanding of the current state and potential of gene therapy for Hemophilia A.

Adeno, Associated Virus Gene Therapy Of Hemophilia A
INTRODUCTION
Gene therapy is the process of new genes
into cells for restoring or adding gene
expression. The main objective of doing
this is to treat a person from many diseases.
In this process, generally a mutated gene is
being replaced by with DNA that encodes a
functional copy. A therapeutic drug is
generated by encoding DNA.
Virus Gene Therapy of Haemophilia Disease Current and Emerging Gene Therapies
Various studies state that haemophilia can
be treated permanently by gene therapy. In
a case adeno, associated virus was used to
deliver a codon-optimized version of
human factor VIII gene. The vector was
administered into three different cohorts of
patients such as low, high and medium
dosage groups. This therapy was managed
and it has no adverse reactions because of
the treatment. Bleeding decreases after the
gene therapy and this allows cessation of
prophylactic FVIII treatments.
REFERENCES
Haemophilia is an X linked disorder that is
characterized by continuous bleeding in
joints and muscles and increase in risk for
intracranial hernorrhage. The main cause of
this disease is deficiency of the functional
plasma clotting factor VIII. This results
from mutations in the FVIII gene. The
Adeno associated viruses of parvovirus
family are small viruses that have a genome
of single stranded DNA. Such kind of virus
is used for inserting genetic material at a
particular site on chromosome with a
certainty of hundred percent. On the other
hand there are few disadvantages of use of
adeno-associated virus is that it includes
small amount of DNA it is capable to carry
and produce.
Current and Emerging Gene
Therapies
Virus Gene Therapy of
Haemophilia Disease
Green, D. and Ludlam, C.A., 2013. Fast
Facts: Bleeding Disorders. Karger Medical
and Scientific Publishers.
Primer, A., 2012. Immunotherapy.
Liu, D., 2017. Hemophilia and Learning to
Love Science.