Recent Articles2022-03-14T07:39:44+00:00
16August, 2022

Treating Cardiovascular Disease by Changing a Single Letter of DNA

Categories: Featured Perspectives On Current Science|

A simple spelling change in liver DNA could improve the future of cardiovascular disease treatment. On July 12, biotechnology company Verve Therapeutics announced that the first patient had received a novel gene editing medicine designed to permanently reduce "bad" LDL cholesterol — the fatty molecule that causes arteries to [...]

26July, 2022

FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients

Categories: Featured Perspectives On Current Science|

In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in the pipeline. As oligonucleotide therapeutics continue to develop as an emerging and promising treatment for rare diseases, the US Food [...]

7July, 2022

A New Conjugated siRNA provides broad delivery in the CNS, Lung, and Eye

Categories: Perspectives on Current Science|

Ever since the groundbreaking discovery of RNA interference and its role as a gene-silencing mechanism in mammalian cells, an ongoing process of discovery has been underway to harness its potential in treating disease. A key mechanism that induces gene-silencing in the RNA pathway is small interfering RNA (siRNA). Although [...]

20June, 2022

Positive Results from Tofersen VALOR Trial and OLE Integrated Data

Categories: Perspectives on Current Science|

People diagnosed with the progressive neurodegenerative disease ALS previously had no hope of an effective treatment that could delay the onset of symptoms or extend their lifespan. However, as a result of recent advances in science and medicine, multiple oligonucleotide therapeutics are being developed to provide treatments, one of [...]

6June, 2022

CRISPR Babies and the Scientist Behind Their Editing

Categories: Perspectives on Current Science|

At an international genome-editing summit in Hong Kong, MIT Technology Review reporter Antonio Regalado uncovered the existence of the CRISPR baby project. The now infamous scientist, He Jiankui, had used CRISPR-Cas9, a genetic engineering tool, to edit the CCR5 gene of three embryos with the goal of making them [...]

31May, 2022

CRISPR Patent Rights and Their Effect on the Industry

Categories: Perspectives on Current Science|

A decade ago, Jennifer Doudna of the University of California, Berkley and Emmanuelle Charpentier of the Max Planck Institute for Infection Biology drafted blueprints for a groundbreaking gene editing technique. The two scientists had found a way to cut and make precise changes to DNA — a discovery that [...]

9May, 2022

Could Artificial Intelligence Provide the Key to Life Saving Medicines?

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

Drug discovery is time consuming and expensive, requiring trial and error screening. This means that many people have to wait decades, or a lifetime, to find a treatment for their disease. Others, especially those with rare diseases, may never have hope of a treatment. However, AI and deep learning [...]

22April, 2022

A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD

Categories: Perspectives on Current Science|

Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body's muscle tissues to progressively waste away until all mobility is lost. Eventually, the heart muscle becomes fatty and fibrotic, typically leading to heart failure and death by age 30. There is no cure, and the approved drugs [...]

9March, 2022

Support for Ukrainian Scientists

Categories: Perspectives on Current Science|

The OTS joins other scientific organizations in seeking to help Ukrainian scientists. Specifically, we invite Ukrainian students, scientists, and industry professionals with interests in the field of nucleic acids and oligonucleotide therapeutics to post their contact information and CVs on the OTS website. We ask our membership to spread [...]

FDA Draft Guidance – An Exciting Step in the Journey to Bring Oligonucleotide Therapeutics to Patients

July 26th, 2022|Categories: Featured Perspectives On Current Science|

In recent years, antisense and small interfering RNA (siRNA) oligonucleotide therapeutics have been FDA-approved to treat rare diseases, and many oligonucleotide therapeutics aimed at treating common chronic diseases are in [...]

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