Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted
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 ...
siRNA: From a Powerful Research Tool to Potent Therapeutic Modality
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 ...
The Astonishing History, Controversies, and Game-Changing Uses of CRISPR
Many of us view CRISPR as an incredibly useful tool. It is even referred to as genetic scissors because CRISPR allows scientists to edit the genes (the DNA) of organisms, from the smallest cells to an entire mammal. Because of its revolutionary impact on life sciences, the discovery of ...
The Emerging Era of N-of-1 Drugs for Ultra-Rare Genetic Diseases
Imagine if a medication or therapy could be created to treat just one individual with an ultra-rare disease. There are nearly 7,000 known rare diseases (1) and many are debilitating and ultimately fatal. In the U.S., a disease is considered rare when it affects as many as 200,000 people. ...
Facts about COVID-19 mRNA Vaccines and the Decades of Research That Went into Creating Them
Posted: February 11, 2021 | Updated: October 8, 2021 Despite the global threat of COVID-19, mRNA vaccines, which are highly effective at preventing COVID-19 illness and have passed rigorous safety standards, have been met with skepticism by many people. This suspicion is due in part to misconceptions and misinformation ...
20th Anniversary of RNA Interference in Mammalian Cells
The groundbreaking discovery of RNA interference by Craig Mello and Andrew Fire was swiftly followed by the discovery of RNAi as a gene-silencing mechanism in mammalian cells. Since these breakthroughs, members of our community have devoted their lives to building on these exciting discoveries, and together we are producing ...
Oligonucleotides and the OTS Community Provide Excellent Solutions to the COVID-19 Pandemic
Oligonucleotide therapies are relatively new in the field of medicine, but they have allowed us to make astounding progress in the fight against COVID-19. Rather than simply repurposing existing drugs or spending years to develop new drugs and vaccines, oligo therapies are quickly providing solutions to this global health ...
Oligonucleotide Therapeutics: New Drugs That Could Stop COVID-19
In response to the COVID-19 pandemic, scientists around the world have joined together in applying their skills and technologies to fight this global threat. Some of the most promising technologies to fight the coronavirus are based on a new type of drug called oligonucleotide therapeutics. In order to promote ...
Coronavirus Highlights Urgent Need for Science-Backed Research
News Release from Mary Ann Liebert, Inc. publishers Coronavirus Highlights Urgent Need for Science-Backed Research As an academic publisher, we publish many important resources relevant to COVID-19 such as Health Security, Viral Immunology, Telemedicine and e-Health, and Vector Borne and Zoonotic Diseases. We are keeping our finger on the pulse ...
Interview with the 2019 OTS Lifetime Achievement Award Winner Muthiah (Mano) Manoharan
Dr. Alan M. Gewirtz Memorial Scholarship Award: Bruno M.D.C. Godinho, PhD, RNA Therapeutics Institute, UMass Medical School
Phase III Trial of ASO Therapy for the Treatment of Huntington’s Disease Halted
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 ...
siRNA: From a Powerful Research Tool to Potent Therapeutic Modality
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 ...
The Astonishing History, Controversies, and Game-Changing Uses of CRISPR
Many of us view CRISPR as an incredibly useful tool. It is even referred to as genetic scissors because CRISPR allows scientists to edit the genes (the DNA) of ...
The Emerging Era of N-of-1 Drugs for Ultra-Rare Genetic Diseases
Imagine if a medication or therapy could be created to treat just one individual with an ultra-rare disease. There are nearly 7,000 known rare diseases (1) and many are ...
Facts about COVID-19 mRNA Vaccines and the Decades of Research That Went into Creating Them
Posted: February 11, 2021 | Updated: October 8, 2021 Despite the global threat of COVID-19, mRNA vaccines, which are highly effective at preventing COVID-19 illness and have passed rigorous ...
20th Anniversary of RNA Interference in Mammalian Cells
The groundbreaking discovery of RNA interference by Craig Mello and Andrew Fire was swiftly followed by the discovery of RNAi as a gene-silencing mechanism in mammalian cells. Since these ...
Oligonucleotides and the OTS Community Provide Excellent Solutions to the COVID-19 Pandemic
Oligonucleotide therapies are relatively new in the field of medicine, but they have allowed us to make astounding progress in the fight against COVID-19. Rather than simply repurposing existing ...
Oligonucleotide Therapeutics: New Drugs That Could Stop COVID-19
In response to the COVID-19 pandemic, scientists around the world have joined together in applying their skills and technologies to fight this global threat. Some of the most promising ...
Coronavirus Highlights Urgent Need for Science-Backed Research
News Release from Mary Ann Liebert, Inc. publishers Coronavirus Highlights Urgent Need for Science-Backed Research As an academic publisher, we publish many important resources relevant to COVID-19 such as Health ...
Interview with the 2019 OTS Lifetime Achievement Award Winner Muthiah (Mano) Manoharan
Dr. Alan M. Gewirtz Memorial Scholarship Award: Bruno M.D.C. Godinho, PhD, RNA Therapeutics Institute, UMass Medical School