2021 Basic Paper Of The Year Award – Winners

October 28th, 2021|Categories: Basic Featured Paper of the Year, Paper of the Year Award|

Investigating the pharmacodynamic durability of GalNAc-siRNA conjugates Givosiran, a GalNAc-conjugated siRNA, which was recently approved on the market, displays sustained knock-down over month-long periods. The following paper investigates the reasons behind those impressive pharmacodynamic properties. After looking into various mechanisms related to stability, accumulation or ...

2021 Mary Ann Liebert Publishers, Inc. Young Investigator Award Winner Alex Garanto, PhD

October 28th, 2021|Categories: Young Investigator Award|

Alex Garanto, PhD Radboud University Medical Center (Radboudumc) Biography: Dr. Alejandro (Alex) Garanto is currently an Assistant Professor at the Departments of Pediatrics and Human Genetics of the Radboudumc in Nijmegen (the Netherlands). He obtained his PhD from the University of Barcelona in 2011 ...

2021 Dr. Alan M. Gewirtz Memorial Scholarship Award for Postdoctoral Fellows and Junior Industrial Professionals Winner Kotaro Yoshioka, MD, PhD

October 28th, 2021|Categories: Dr. Alan M. Gewirtz Memorial Scholarship|

Kotaro Yoshioka received his PhD in 2016 from Tokyo Medical and Dental University (Japan), where he has developed a unique double-stranded ASO technology, “Heteroduplex oligonucleotide (HDO)” in the laboratory

2021 Dr. Alan M. Gewirtz Memorial Scholarship Award for Graduate Students Winner Mr. Hassan Fakih

October 28th, 2021|Categories: Dr. Alan M. Gewirtz Memorial Scholarship|

Mr. Hassan Fakih Department of Chemistry, McGill University Biography: Hassan obtained his bachelor’s degree in chemistry from the American University of Beirut in 2016. Then, he Joined the Sleiman Lab at the Chemistry department of McGill University to pursue his doctorate degree. His work ...

New Miniature CRISPR-Cas Systems Engineered to Function in Human Cells

October 28th, 2021|Categories: Perspectives on Current Science|

CRISPR-Cas systems provide a revolutionary approach to curing disease through precise genome editing and CRISPR therapies have delivered promising early successes. However, engineering more highly efficient, compact Cas systems could result in a more versatile tool that could greatly improve and expand CRISPR-based therapeutics. As ...

President’s Pick of Presentations from the Annual OTS Meeting

October 26th, 2021|Categories: Perspectives on Current Science, Presidents Pick|

By: Annemieke Aartsma-Rus, Ph.D. The second virtual annual meeting of the Oligonucleotide Therapeutics Society (OTS) took place September 26-29 on a computer or laptop near you. Despite the challenges the pandemic poses on research and development in general, the oligonucleotide therapy field has made ...

Non-Profits Create Next-Generation Medicines that Treat Ultra-Rare Diseases

October 8th, 2021|Categories: Perspectives on Current Science|

Hundreds of millions of people worldwide are living with a rare disease. Most rare diseases do not currently have a cure. So, once diagnosed with a rare disease, most patients and their families are merely given strategies to manage the disease symptoms and try ...

Interview with Erin M. McConnell, PhD (Chemistry)

October 8th, 2021|Categories: Featured Members|

My introduction to the field was through functional nucleic acids called aptamers – small pieces of DNA that act as molecular recognition tools. I learned about them as an undergraduate student while completing my B.Sc. in neuroscience and chemistry at Carleton University, from Prof. Maria C. DeRosa, who would become my Ph.D. supervisor.

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