Society News2021-08-27T10:57:04+00:00
19January, 2022

Does CRISPR Gene Editing Increase the Risk of Developing Cancer?

January 19th, 2022|Categories: Featured 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, as we must optimize CRISPR technology while we are also [...]

1January, 2022

Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease

January 1st, 2022|Categories: Featured Perspectives On Current Science|

There is currently no treatment to slow or halt disease progression, but proactive, comprehensive medical care is helpful for those diagnosed with the disease. Various interventions such as physiotherapy, speech therapy, nutrition, and anti-epileptic drugs can help patients maintain the best possible quality of life as the disease progresses. The disease is almost always caused by missense mutations in [...]

1December, 2021

Can an ASO Reduce Disease Progression in ALS?

December 1st, 2021|Categories: Perspectives on Current Science|

What if you noticed your body acting a bit “off,” doing something unusual and more than a bit worrying? Then, you go to a doctor and are eventually told that you have a debilitating neurodegenerative disease and can expect to progressively lose control of your muscles. Your quality of life will rapidly decline, and death is likely within two [...]

10November, 2021

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 – a platform that could allow for administration of siRNA medications just once a year with highly potent knockdown of target mRNA. This platform is being tested in preclinical studies [...]

28October, 2021

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 Dr. Stanley Qi pointed out, rather than viewing CRISPR as genetic scissors, it may [...]

26October, 2021

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 the pandemic poses on research and development in general, the oligonucleotide therapy field has made many advances, including the first systemic trial for genome editing and the delivery of 2 [...]

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