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The quest for an HIV vaccine has been given a bad prognosis recently, due to increasing agreement that the human immune system isn't clever enough to outsmart the ever-changing surface of the virus. But now a new approach promises to solve the problem by sidestepping the immune system altogether, instead using gene therapy to produce immune molecules that neutralize the virus.
To tackle this problem, Johnson turned to another virus for help. A modified version of the adeno-associated virus (AAV) has been proved to work as a convenient vector for delivering gene snippets safely into the human body for gene therapy, and it has been used successfully to treat hemophilia and congenital blindness by supplying patients with genes that are otherwise missing.
Originally posted by Terapin
The quest for an HIV vaccine has been given a bad prognosis recently, due to increasing agreement that the human immune system isn't clever enough to outsmart the ever-changing surface of the virus. But now a new approach promises to solve the problem by sidestepping the immune system altogether, instead using gene therapy to produce immune molecules that neutralize the virus.
Gene transfer offers HIV hope
This approach has been put to use in trials against a simian (monkey) virus SIV, very similar to HIV and it worked!!!! Gene therapy is a new approach to the problem and it could be one that finally solves the problem for AIDS and a number of other viral diseases.
Much like with stem cell research, gene therapy is not without some controversy, particularly with the religious crowd, but the science behind it is quite solid. By attacking viral diseases at the genetic level, we can surely gain the upper hand. This is good new indeed!
Is it worth it? You tell me.
Adeno-associated viruses, from the parvovirus family, are small viruses with a genome of single stranded DNA. The wild type AAV can insert genetic material at a specific site on chromosome 19 with near 100% certainty. But the recombinant AAV, which does not contain any viral genes and only the therapeutic gene, does not integrate into the genome. Instead the recombinant viral genome fuses at its ends via the ITR (inverted terminal repeats) recombination to form circular, episomal forms which are predicted to be the primary cause of the long term gene expression. There are a few disadvantages to using AAV, including the small amount of DNA it can carry (low capacity) and the difficulty in producing it. This type of virus is being used, however, because it is non-pathogenic (most people carry this harmless virus). In contrast to adenoviruses, most people treated with AAV will not build an immune response to remove the virus and the cells that have been successfully treated with it. Several trials with AAV are on-going or in preparation, mainly trying to treat muscle and eye diseases; the two tissues where the virus seems particularly useful. However, clinical trials have also been initiated where AAV vectors are used to deliver genes to the brain. This is possible because AAV viruses can infect non-dividing (quiescent) cells, such as neurons in which their genomes are expressed for a long time.
Clinical trials
Data on safe and long-lasting rAAV-mediated transgene expression in organs of animal models of human disease such as lung, liver, central nervous system and eye, together with improvements in vector production and purification methods provided the rational for initiating clinical studies with rAAV vectors. Currently, these clinical trials are either in phase I or in phase II. The former studies aim at determining safety and often also maximum tolerable dose of the therapeutic agent, while the latter entail the assessment of its efficacy and have higher statistical significance to detect potential side effects. Ailments being targeted include Parkinson's disease, Canavan's disease, α1-antitrypsin deficiency, cystic fibrosis (cystic fibrosis transmembrane conductance regulator [CFTR] deficiency) and hemophilia B (blood clotting factor IX [FIX] deficiency). Cystic fibrosis and hemophilia B are two examples of which more information is available. In fact, more than one decade ago, cystic fibrosis patients were the first human individuals subjected to rAAV administration [106].
At the only hospital in the capital of this tiny West African nation, a 3-year-old AIDS patient named Suleiman receives his daily dose of medication -- a murky brown concoction of seven herbs and spices served out of a bottle that once contained pancake syrup.
The boy is told a spoonful a day will make him better. His mother, Fatuma, takes the same concoction, as do several dozen other AIDS and HIV patients here. Adults take two spoonfuls.
"My uncle raped me when I was 14. I was arguing with him and he hit me. I blacked out and when I woke up I was in hospital and the police told me I had been raped. My uncle had HIV. After he attacked me he ran away. Later he wrote me a letter asking for forgiveness. He said he raped me because he thought having sex with a virgin would cure him of his disease. He said he felt sorry for me because now I had HIV and I was going to die. Then he killed himself. When my mother died in 1998 my auntie sent me money to come from the Eastern Cape and live with her in Cape Town. I was going to school and everything was good for a while but then I got sick. I told my auntie what had happened but she didn't believe me, she didn't believe that my uncle had infected me, she said I must have caught HIV from my boyfriend.