Recent Articles2022-03-14T07:39:44+00:00
19January, 2022

Does CRISPR Gene Editing Increase the Risk of Developing Cancer?

Categories: Perspectives on Current Science|

CRISPR is a useful tool in a wide array of applications, and one of the most headline-grabbing is its potential in gene editing. As with any new technology or medication, there are inherent risks that must be discovered and mitigated. This is especially true in the arena of gene-editing, [...]

1January, 2022

Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease

Categories: Featured Perspectives On Current Science, Perspectives on Current Science|

It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs have proven to be efficient at providing deep reductions in disease causing proteins, and a treatment that produced significant results [...]

24December, 2021

Looking back… Looking forward

Categories: Blog|

This will be my last contribution that I write as President of our Society. On January 1st, 2022, I will become ‘Immediate Past-President’. David Corey (University of Texas, Southwestern) will take over as President and Richard Geary (Ionis Pharmaceuticals) will become the Society’s next President-Elect. As we delayed this [...]

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: Presidents Pick, Perspectives on Current Science|

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 [...]

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, [...]

IKARIA – An Innovative Platform that Yields Long-Acting siRNA

November 10th, 2021|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 [...]

Comments Off on IKARIA – An Innovative Platform that Yields Long-Acting siRNA

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 [...]

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

President’s Pick of Presentations from the Annual OTS Meeting

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

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 [...]

Comments Off on President’s Pick of Presentations from the Annual OTS Meeting

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 [...]

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

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 [...]

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

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