Recent Articles2021-11-04T17:51:22+00:00
10November, 2021

IKARIA – An Innovative Platform that Yields Long-Acting siRNA

Categories: Perspectives on Current Science|

In this year that marks the 20th Anniversary of the discovery of RNA interference as a gene-silencing mechanism in mammalian cells, Alnylam announced another remarkable development in siRNA therapeutics – a platform that could allow for administration of siRNA medications just once a year with highly potent knockdown of [...]

28October, 2021

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

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 Dr. Stanley Qi pointed [...]

26October, 2021

President’s Pick of Presentations from the Annual OTS Meeting

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 many advances, including the first systemic trial [...]

8October, 2021

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

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 to minimize the impact on [...]

13September, 2021

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

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 Tokyo Medical and Dental University, Takeda [...]

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 [...]

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