Recent Articles2021-11-04T17:51:22+00:00
21June, 2021

Can Oligonucleotides Target RNA Splicing to Treat Disease?

Categories: Perspectives on Current Science|

The human body is phenomenally complex, and we are continually discovering incredible facts about it. One fascinating aspect lies in splicing of RNA as it is processed in the cell. Alternate splicing of a single pre-mRNA molecule can create tens of thousands of different mRNAs, which contributes to the [...]

29May, 2021

mRNA Is Capable of Providing Solutions Far Beyond Vaccines

Categories: Perspectives on Current Science|

When the FDA issued emergency use authorization for Pfizer-BioNTech and Moderna’s COVID-19 vaccines, mRNA instantly became a household word. Many people believed that this new technology had sprung up out of nowhere in less than a year. What they did not know is that it had been in development [...]

20May, 2021

Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

Categories: Perspectives on Current Science|

Imagine if your muscles began weakening for no apparent reason. Then, after an ongoing search for a diagnosis, you learn that you have a disease in which your muscles would progressively waste away and become weaker until you would no longer be able to walk. Ultimately, the disease would [...]

14April, 2021

Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted

Categories: Perspectives on Current Science|

Recently, people who have been impacted by Huntington’s disease, their families, and the OTS community were disappointed to learn that the GENERATION HD1 Phase III study of Tominersen was halted based on the recommendation of an unblinded Independent Data Monitoring Committee. Although not based on emergent safety concerns, the specific [...]

30March, 2021

siRNA: From a Powerful Research Tool to Potent Therapeutic Modality

Categories: Perspectives on Current Science|

Earlier this year we celebrated the 20th Anniversary of RNA Interference in Mammalian Cells. In this article, we are going to discuss small interfering RNA (siRNA), a key mechanism that induces gene silencing in the RNAi pathway. siRNAs are highly specific, making them a powerful tool to suppress the [...]

Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

May 20th, 2021|Categories: Perspectives on Current Science|

Imagine if your muscles began weakening for no apparent reason. Then, after an ongoing search for a diagnosis, you learn that you have a disease in which your muscles [...]

Comments Off on Oligonucleotides Provide Hope for the Treatment of Duchenne Muscular Dystrophy

Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted

April 14th, 2021|Categories: Perspectives on Current Science|

Recently, people who have been impacted by Huntington’s disease, their families, and the OTS community were disappointed to learn that the GENERATION HD1 Phase III study of Tominersen was halted [...]

Comments Off on Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted

siRNA: From a Powerful Research Tool to Potent Therapeutic Modality

March 30th, 2021|Categories: Perspectives on Current Science|

Earlier this year we celebrated the 20th Anniversary of RNA Interference in Mammalian Cells. In this article, we are going to discuss small interfering RNA (siRNA), a key mechanism [...]

Comments Off on siRNA: From a Powerful Research Tool to Potent Therapeutic Modality
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