Disadvantages Of Adeno-Associated Viruses

Cystic fibrosis is known to be one of the most common and deadly diseases in Caucasians, affecting 1 in 2500 children. This percentage results in 30,000 individuals within the United States to be diagnosed with CF. There are over 1900 mutations of this gene that cause a wide variety of severities within this disease. (McCance, Huether, Brashers, & Rote, 2010) Due to its complex mutation and unknown cause, only treating the symptoms of CF have been the main treatment protocol to this disease. Current treatments are cumbersome and expensive providing patients with life expectancy only into their twenties, but usually younger in most cases. There has been specific progress towards a cure involving gene therapy providing hope for a cure to

This article covers the Seneca Valley Virus (SVV-001) as a hopeful for an oncolytic treatment of certain cancer types. More specifically those with neuroendocrine properties such as rhabdomyosarcoma, Wilms tumor, glioblastoma, neuroblastoma, and adult small-cell lung cancer. Each of which effect smooth/skeletal muscle cells, kidneys/adrenal glands (mainly in children), astrocytes of the brain, nerve cells of a fetus, and lung cells in adults respectively. The virus was discovered by accident in a contaminated cell culture that contained bovine serum to promote growth. The virus was later discovered to be almost exclusively found in farm animals such as cows and pigs, due to the presence of neutralizing antibodies that were later to only ever have been found in one human sample. Just as important as that, the virus only targets the cells of the above-mentioned cancers/tumors, is a self-replicating RNA virus, and its inability to infect other cells in the body all come together to result in the lysis of these specific cancer cells. These properties alone give great hope for SVV-001 as a treatment for those infected by these diseases, and prompted for more research into its medicinal possibilities.

viruses, and Herpes simplex viruses” (2). The primary virus used in gene therapy is the “adenoassociated

“A team of scientists conducted a trial therapy that replaced defective genes that cause cystic fibrosis. The results showed noteworthy advantage that enhanced patients' lung function. This innovation was developed by the technology firm Imperial Innovations. The worn-out genes were replaced by using inhaled molecules of DNA, which send normal working duplicate of the gene to lung cells. "Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group," said Eric Alton, the team's lead researcher, who works at Imperial College London.”

Once this is determined, we will use gene therapy to treat Cystic Fibrosis.  We will use the adenovirus treatment mentioned above, but in order to counter the fact that it doesn’t last long enough, we will propose more frequent dosages of this treatment.  The dosages will be administered with aerosol.  We will have to do research to find the right amount of time to wait in between dosages because if we give them too close together, this will cause too much inflammation.  If this approach does not end up working, we will also consider finding a way to enable the adenovirus once it has been administered.  This would allow the adenovirus to replicate and stay in the patient’s DNA, which could possibly cure CF.  Another option is finding a way to lessen the cell-mediated immune response.  Once we performed research and clinical trials, we would determine which combinations of these options would work to cure Cystic

Trying to find a cure is hard some virus can help but are they to deadly. Scientist are saying that this virus helps but the immune system does not allow it. The Lassa-VSV virus carries a gene that causes it to produce a fluorescent protein that helps researchers track which

One side to this issue is the use of viral vectors in gene therapy to cure the underlying disease. One of the first viral vectors used was the adenovirus vector. The adenovirus by itself is a linear double-stranded DNA molecule that causes mild respiratory infections, but when used as a vector, certain genes and regions are removed to make it less harmful (Alton, et al. 2010). The adenovirus vector is easy to grow, adaptable, able to infect both dividing and non-dividing cells and is quick to

Adenoviruses are the cause of a wild range of bad illnesses such as common, sore throat,

Genetic engineering allows biologists to manipulate an organism's genomes by utilizing biotechnology (Moulton, 2004 ). The Talimogene Laherparepvec virus, otherwise known as T-vec, is an oncolytic virus. A oncolytic virus, is a virus that destroys cancerous cells. The T-vec virus has recently had successful results for treating melanoma cancer. The virus originated from the herpes simplex virus 1 and has been genetically altered to help destroy cancer cells.The virus gets injected into the lesions, lesions are areas on or in the body that have been damaged . Not only does the virus destroy cancer cells, it also activities the immune system to attack the cancer cells. Many changes were done to diminish the virus, expand the selective for the cancer cells, and release the cytokine. The modifications that were implanted in the virus were, HSV-1 strain (JS1) which is the DNA set of the herpes virus, that increases the death of the tumour cells. To delete ICP34.5, that stops the HSV infection in the cells that are not tumorous, and will provide selective tumour duplication. As Well as the deletion of ICP47 that allows antigen presentation, an antigen is a substance that helps enable an immune response. Placing in the US11 will

Gene therapy is a way in which an individual with a preexisting genetic disorder (in this case ADA deficiency) can be treated. This involves the insertion of a functioning ADA gene into an individual’s cells through the use of viral vectors, to replace the mutated ADA gene that is non-functioning or functioning incorrectly. In order for this process to be viable multiple techniques are used, including: restriction enzymes, ligation, PCR and transfection. Through these processes an individual is able to

Filoviruses are membrane-enveloped filamentous viruses that contain a negative sense single- stranded RNA. Filoviruses possess single-stranded, negative-sense RNA genomes of approximately 19 kilobases. These genomes have seven distinct genes, each of which functions as a separate transcriptional unit, coding for eight proteins. These seven open reading frames encode structural proteins, including the virion envelope glycoprotein (GP), nucleoprotein (NP), and matrix proteins VP24 and VP40; nonstructural proteins, including VP30 and VP35; and the viral polymerase. The GP open reading frame of Ebola virus gives rise to two gene products, a soluble 60- to 70-kDa protein (sGP) and a full-length 150- to 170-kDa protein (GP) that inserts into the

The greatest challenge faced when attempting to cure Cystic Fibrosis through gene therapy is the use of a vector that will successfully transport the correct CFTR gene to restore lung functionalty. Across the sources the debate of viral vectors against non-viral vectors is dominant as viral vectors are show to have a

Cystic Fibrosis (CF), is another disease that is taking to genetic therapy. If a corrected gene could somehow enter the cells that line the lungs, it will then start producing the critical proteins that CF patients need. This has been done, although in small quantities. These results, however, have raised hopes that sometime in the future, CF may be curable.

It has been clinically observed that an increase in 1-2% in the circulating levels of the deficient clotting factor can significantly enhance the bleeding diathesis, therefore the therapeutic goal for gene therapy is essential. Currently intravenous infusions of either recombinant or plasma derived factor VII or factor IX proteins plasma transfusions are the treatment option for haemophiliacs (coagulation disorder paper). The therapeutic goal for the coagulation factor gene therapy is to deliver a normal copy of the gene to produce efficient levels in plasma for normal coagulation to occur in place of a deficient or dysfunctional protein as a result of a germline mutation. Researchers have discovered that the vector system for coagulation therapy is adeno-associated virus (AAV) based strategies as they have shown to be leading the way for coagulation therapy. AAV are infectious agents that belong to the parvovirus family and result in asymptomatic infection in humans. To date, about 31 Haemophilia B patients have been treated in 5 AAV clinical trials. A clinical study to deliver factor VIII has only just one patient enrolled. As AVV viral vectors have small single stranded DNA with a genome of 4.7Kb this has presented a limitation in the introduction of factor VIII transgenes as the factor

“Helper-dependent adenoviral vectors (HDAds) possess long homology arms that mediate high-efficiency gene editing. These long homology arms may permit simultaneous introduction of multiple modifications into a large genomic region or may permit a single HDAd to correct many different individual mutations spread widely across a gene” says Palmer. “HDAds can efficiently deliver foreign DNA into the nucleus of target cells. Furthermore, they are deleted of all viral-coding sequences, thereby reducing their toxicity and increasing their cloning capacity to 37 kb.” Twelve 2 bp insertions were made into an HDAd by this team of scientists, in order to determine how much of the modified gene-of-interest had been introduced into the CFTR locus after homologous-recombination. Of the 83 targeted recombinants which were analyzed, the data revealed that even markers a good distance from the desired marker (11 kb, the farthest being 22 kb) were introduced to the target gene efficiently and at high frequencies (about 21.7%). The results of this experiment are consistent with the idea that HDAds can make changes over large genomic regions, and may prove useful in the advancement of individual treatment for genetic disorders such as Cystic Fibrosis.