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The Dire Need for Novel Research Tools

14/9/2019 10:19:51 AM

In translational medicine, the process of bringing a drug candidate from the bench into the clinic is a lengthy one. As researchers progress their studies from cells into animals, new and novel modalities and methods are needed to solve challenges and discover new mechanisms associated with the complexity of translational medicine.

Challenges with Primary Cell & Stem Cell:

One of the first challenges researchers may face is dealing with patient derived primary cells, often times immune or stem cells. Primary cells, often diseased donor cells, can be very tricky to work with and are very difficult to transfect. Traditional gene knockdown techniques such as RNAi and CRISPR methods may not work as efficiently as before. Often times, the researcher runs into toxicity issues and low cell viability, as a result of minimally effective methods, often associated with toxicity and off target effects. Working in primary cells may be one of the first steps a researcher may take to gradually advance their research from benchtop to the clinic.

At AUM BioTech, we provide novel research tools to scientists and researchers facing some of these challenges, and effectively solve them using our next generation antisense technology. Often times, siRNA and CRISPR techniques are completely ineffective when dealing with novel primary and immune cells.

Just last year, Dubielecka et al. published an article in Blood, in which the authors knockdown ABI1 in patient derived hematopoietic progenitors and granulocytes, often a daunting task with traditional knockdown techniques, as these cells are very difficult to transfect and very sensitive to transfection reagents.

The authors could see up to 95% knockdown (without the use of any delivery agent or transfection agent) - which is incredible. Kindly, review Figure 4H and Supplementary Figure 6A to review the knockdown data. Further, very nice cellular uptake can also be seen in these cells (Supplementary Figure 6B-C). Kindly also review Figure 4I and Supplementary Figure 6D.

ncRNA with FANA

Further, FANA ASOs are also helping advance genetic discovery in fields that are just now being discovered. Namely, the ncRNA field is an emerging field that is being further discovered on a daily basis. ncRNAs are proving to be a promising field in precision and translational medicine, and are now a focal point of cancer research.

Our Experts for Stem Cell & Immune Cell

Dr. Leo Kurian’s work involves discovery of a currently unknown class of genes called ying yang lncRNAs (yylncRNA). Dr. Kurian’s recent work indicates that yylncT is essential for mesoderm differentiation of human pluripotent stem cells.

According to Dr. Kurian, “One of the challenges we faced during this project was to find an effective method to knock-down nuclear localized transcripts, since yylncT is near-exclusively nuclear. We tested multiple ASOs with minimal success and then we came across the FANA-technology. In our hands, FANA gave us the most consistent knock-down of nuclear transcripts.”

Kindly find Dr. Dubielecka’s and Dr. Kurian’s publications on ABI1 and yylncRNA, respectively, here, along with some of our other studies : https://www.aumbiotech.com/Publications

FANA ASOs have proven to be a valuable research tool in genetics, oncology, and RNA research. Through robust activity in primary and immune cells, and in the ncRNA field, FANAs allow robust experimental data, often times impossible with traditional RNAi and CRISPR methods.