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.

Oligonucleotides Safely Cross the Blood-Brain Barrier to Knock Down Gene Expression

September 13th, 2021|Categories: Perspectives on Current Science|

Neurodegenerative diseases, brain tumors, and infectious diseases and inflammatory conditions that impact the brain wreak havoc on a person’s health and are notoriously difficult to treat. Oligonucleotide drugs could provide solutions to many pathological brain conditions if certain challenges could be overcome. Researchers at ...

Ligand Conjugation – Expanding the Reach of Oligonucleotide Therapeutics

August 25th, 2021|Categories: Featured Perspectives On Current Science|

Oligonucleotide therapies possess enormous potential as healing, life-changing, and cancer-curing drugs. However, there are many barriers to utilizing oligonucleotides to treat disease. Discovering or creating effective delivery methods for each target cell or tissue type solves many of these problems and there are multiple ...

OTS President’s Paper Pick – August 2021

August 9th, 2021|Categories: Perspectives on Current Science, Presidents Pick|

By: Annemieke Aartsma-Rus, Ph.D. The paper information: CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis Julian D Gillmore, Ed Gane, Jorg Taube, Justin Kao, Marianna Fontana, Michael L Maitland, Jessica Seitzer, Daniel O’Connell, Kathryn R Walsh, Kristy Wood, Jonathan Phillips, Yuanxin Xu, Adam Amaral, Adam ...

Lipid Nanoparticles: Nanomedicine’s Triumph

August 6th, 2021|Categories: Perspectives on Current Science|

Oligonucleotide therapeutics have been making headlines this past year, between the groundbreaking new mRNA vaccines and the recent news of phenomenal interim results for Intellia’s investigational genome editing treatment for ATTR amyloidosis. Neither of these would have been possible without a lesser-known component – ...

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