Gene Therapy for Blood Genetic Diseases

RNAi-based gene therapy (RNA interference) is quickly becoming a hot topic in the world of therapies for blood diseases. Blood diseases are those defined as disorders in the hematopoietic system or plasma compounds. Therapeutic approaches for these diseases are divided into several categories, including chemotherapy, radiotherapy, hematopoietic stem cell transplantation, and RNAi-based gene therapy. Of these, RNAi-based gene therapy is progressively becoming an alternative offering the possibility of a permanent cure for some blood diseases.

Gene therapy attempts to treat inherited diseases using normal copies of the defective genes to correct a cellular dysfunction or provide a new cellular function. RNAi not only suppresses transcription by transcriptional gene silencing but it also activates a homology-based mRNA degradation process by post-transcriptional gene silencing, both of which result in the decrease of the coding transcript level (mRNA).

RNAi-based gene therapy possesses several therapeutic advantages, including less immunogenicity. This is in large part due to using non-protein-coding "gene products" to trigger RNAi, making gene therapy potentially less likely to be hindered by the host immune system. Another potential advantage is sequence specificity, which, when compared to traditional small molecules and protein drugs, in combination with the universal treatment spectrum make it an ideal treatment for blood genetic diseases.

One example of the advantage of RNAi-based gene therapy is seen through research conducted with myeloid leukemia. Many therapies have been explored as a cure for this disease, of which chemotherapy is always considered a frontline treatment, mainly containing cytotoxic agents and therapeutic molecules. Although the leukemia cells initially respond well to chemotherapy, it tends to lose some effectiveness after about 6-12 months. In addition, side effects of traditional cytotoxic agents arise, which limit its function with the progression of the disease. RNAi-based gene therapy has been explored by researchers in suppressing the growth and proliferation of myeloid leukemia cells. The results showed a significant decrease in the level of target proteins by limiting the expression of certain genes. RNAi-based gene therapy, when compared to treatments such as chemotherapy, would have a significant effect on the cells with the potential for a notable decrease in side effects.

Although a very exciting possible form of therapy for these types of diseases, there is much research to be done before determining whether RNAi-based therapeutics would be an efficient tool. It seems though that the therapy, in combination with other therapies, could be a very effective new way to treat blood genetic diseases.

Genetics in Forensic Odontolgy

Anyone who knows me knows that I am absolutely obsessed with forensics, which is why I decided 10 years ago that I was going to make it my life. I decided that I was going to study forensics in college (even though I hadn't even made it to high school yet), and that I was going to make a career out of it. I typically focus on things dealing with DNA or trace analysis, but I found an interesting article connected with a different branch of forensics: forensic odontology. Forensic odontology, or forensic dentistry, is branch of forensic medicine in which teeth are used in the identification of victims when their bodies are unrecognizable.

Genetic material is obtained in different ways when comparing living suspects to deceased victims. When dealing with living suspects, genetic material is typically obtained through blood or a cheek swab. On the other hand, in order to help determine the identity of a deceased person, the method of obtaining genetic material is a bit more complicated. When it comes to verifying a familial relation involving post-mortem material, time from death and corpse condition play a significant role in the method used. In more recent deaths, soft tissue is a viable option, whereas in situations of longer periods of time, the soft tissue is no longer a suitable option and sources such as bone and teeth are used.

Teeth are a great source of genetic material as natural teeth are the most durable organs in the bodies of vertebrates and they have significant tissue resistance against external injuries. The dental pulp presents a better condition for DNA extraction than other soft tissues as it is protected by the tooth structure. The genetic material extracted from the victim is then compared to either the genetic material obtained from a presumed family member or from a known DNA profile of the victim.


Sources:

http://medind.nic.in/jal/t12/i1/jalt12i1p55.pdf

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3612186/