Schreiber Science: HIV/AIDS being cured on a molecular scale

HIV, or human immunodeficiency virus, is a major health concern across the world due to the risk of the development of AIDS, or acquired immunodeficiency syndrome, if left untreated. Despite the many advances made in the scientific community in the past few decades regarding developing an understanding of HIV and its prevention, there is still no definitive cure.

However, the development of effective treatments for HIV, particularly using RNA-based gene therapy, has become increasingly popular with new technologies and techniques on the genetic level. RNA is a molecule similar to DNA found in all cells and is essential to the production of various structures such as proteins as well as any actions, which take place in a cell.

Using gene therapy, or the artificial manipulation of the genetics of this RNA, can allow it to treat a variety of diseases, HIV included.

The most common treatment for HIV is known as HAART which is a combination therapy involving a large variety of drugs. Despite its effectiveness in controlling the disease progression in HIV-infected individuals, HAART is not a cost-efficient and therefore is not a realistic solution for the treatment of HIV, as it cannot be used to a large degree to treat those in third-world countries where the virus is most prevalent.

Scientists are currently working on treatments which will better account for issues surrounding cost-efficiency and widespread availability, which in recent years has lead to in depth studies regarding RNA-based therapies.

Ribozymes are small RNA molecules with catalytic abilities in specific reactions, making them a viable tool in targeting HIV.  The ribozymes are engineered to provide them with removing genes of interest from a virus.

Before discussing the influence of various genetic therapies on HIV, it is important to understand HIV’s lifecycle. The genetic material of the virus enters the host cell and inserts itself into the host’s DNA in order to cause it to produce more viruses.

This new method of treating HIV results in a lower chance of mutation and less genetic damage in cells infected by the virus.

The first clinical study utilizing ribozymes to target the realm of viral disease in its entirety was conducted on HIV-infected individuals in 1996.

The hepatitis delta virus ribozyme, or HDV ribozyme, is derived from an RNA species found in human cells. This enables the ribozyme to function in the presence of human proteins.

The functions of the HDV ribozyme provide many advantages in engineering to specifically target a viral RNA; however, the HDV ribozyme’s mechanism to recognize a target is not specific enough to treat disease.

The various studies, which have risen to prevalence in recent years, utilize a wide range of RNA-based genetic factors to target viral genes and host dependency factors, providing hope for more effectively treating HIV, along with the possibility of curing the virus in years to come.

Ribozymes, along with various other RNA-based therapies, have proven to be serious candidates for consideration as future treatments of HIV to rival HAART and other present-day treatments.