All of us wants a happy & comfortable life, without any ailments. All of us wants to be active throughout our lifetime. All of us wants to live a life of our choice.
Most of these wishes remains just as wishes for most of us because of various reasons. One reason can be a possible diseases which can turn our life upside down. Hereditary diseases are something which in most case doesn't have a reason to occur or which isn't occurring due to our actions and they are expensive to treat & hard to diagnose.
There are various types of Genetic linked diseases like;
Single gene inheritance which includes cystic fibrosis, sickle cell anemia, Marfan syndrome, Huntington's disease, hemochromatosis etc.
Multifactorial inheritance which includes heart disease, high blood pressure, Alzheimer's disease, arthritis, diabetes, cancer, obesity etc.
Chromosome abnormalities like Turner syndrome, Klinefelter syndrome, Cri du chat syndrome, or the "cry of the cat" syndrome etc.
Mitochondrial inheritance like Leber's hereditary optic atrophy, myoclonic epilepsy with Ragged Red Fibers, mitochondrialencephalopathy, lactic acidosis and stroke-like episodes etc.
As most of the genetic diseases are hard to treat with general drug therapies or surgical procedures, we need to consider something which is highly advanced, more practical & affordable system of management for these disorders, which include diagnosis, treatment & prognosis.
Diagnosis : There are various methods to diagnose a disease. Genetic sequencing is one of the most advanced & accurate method used for determining genetic variations including mutations. But the problem with Sequencing is that it is a very expensive process. And like any other methods of diagnosis, it is also not 100% accurate. There can be a minimal chances of errors or false positives or false negatives.
As the technology is going through rapid advancement & breakthrough discoveries, we now have some very accurate & less expensive methods like Whole Exome Sequencing used in products like B-EDWIG & others. With products like B-EDWIG, an individual can now identify any possible genetic variations in his/her DNA which can lead to a disease in the future for that individual or for his/her generation to come.
Treatment : As our diagnostic methods improves exponentially, the treatment methods are also undergoing some revolutionary advancements. The best treatment methods in development or is already available now for genetic disorders includes gene therapy and DNA editing.
There are various methods for DNA editing including;
Transcription activator-like effector nucleases method. Transcription activator-like effectors (TALEs) can be engineered to bind to practically any desired DNA sequence, so when combined with a nuclease, DNA can be cut at specific locations.
Zinc-finger nucleases method. Zinc finger domains can be engineered to target specific desired DNA sequences and this enables zinc-finger nucleases to target unique sequences within complex genomes. By taking advantage of endogenous DNA repair machinery, these reagents can be used to precisely alter the genomes of higher organisms.
CRISPR method. CRISPR is an abbreviation of Clustered Regularly Interspaced Short Palindromic Repeats. It has a huge potential in treating genetic disorders because of it's precise cutting process.
What we are trying to develop;
At B-Aegis we are working hard on developing a precise & potent DNA editing technology which is based on the Clustered Regularly Interspaced Short Palindromic Repeats from Prevotella and Francisella 1 , the B-CRISPR/Cpf1 method.
Why we are considering Cpf1 rather than Cas9?
Cas9 requires two RNA molecules to cut DNA while Cpf1 needs one. The proteins also cut DNA at different places, offering us more options when selecting an editing site. Cas9 cuts both strands in a DNA molecule at the same position, leaving behind ‘blunt’ ends. Cpf1 leaves one strand longer than the other, creating 'sticky' ends that are easier to work with. Cpf1 appears to be more able to insert new sequences at the cut site, compared to Cas9.
We have demonstrated Multiplex gene editing by B-Cpf1 using a single trans-activating crRNA array in the lab, we have edited four genes in mammalian cells & we are focusing on developing the method in much wider scale.
As biotechnology is creating a better future for everyone, we would like to be a part of it. We are exploring ways to contribute to the ongoing biotechnology revolution, we are working on improving lives. As science is shaping a better life for everyone, there's plenty of hope for the humanity. We'll for sure be shaping our on life the way we want it in the near future!