A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD
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 each help a small portion of patients and cannot target ...
Could a Single Oligonucleotide Medication Treat Multiple Different Diseases?
Our relatively new field is swiftly growing, and another exciting novel therapeutic RNA modality is now gaining traction. tRNA is vital in protein synthesis and by engineering tRNA molecules...
Support for Ukrainian Scientists
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 the word about this initiative to our Ukrainian colleagues. CVs ...
A New ASO Shows Promise in Treating Dravet Syndrome, a Severe Form of Epilepsy
 Typically, a single copy of a gene is enough to support normal growth and development but in a small subset of genes, the loss of one copy can cause serious diseases that are often difficult to treat. One of these conditions, Dravet Syndrome, has no treatment options that directly target the cause of the disease. However, new research from Stoke ...
A Potent ASO Shows Encouraging Results in A Single-Patient Pilot Study for ALS
 Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disease in which motor neurons die. Since the motor neurons that die control voluntary muscles, the disease causes great difficulty for those who must live with it. They will experience loss of motor control of the arms and legs which causes them to frequently trip and fall, drop things, and have cramps ...
Does CRISPR Gene Editing Increase the Risk of Developing Cancer?
 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 forging ...
A Novel Cocktail Drug Penetrates Heart Muscle and May Treat Nearly Half of All Patients with DMD
Duchenne muscular dystrophy (DMD) is a devastating disease that causes the body's muscle tissues to ...
Could a Single Oligonucleotide Medication Treat Multiple Different Diseases?
Our relatively new field is swiftly growing, and another exciting novel therapeutic RNA modality is now gaining traction. tRNA is vital in protein synthesis and by engineering tRNA molecules...
Support for Ukrainian Scientists
The OTS joins other scientific organizations in seeking to help Ukrainian scientists. Specifically, we invite ...
A New ASO Shows Promise in Treating Dravet Syndrome, a Severe Form of Epilepsy
 Typically, a single copy of a gene is enough to support normal growth and development but ...
A Potent ASO Shows Encouraging Results in A Single-Patient Pilot Study for ALS
 Amyotrophic lateral sclerosis (ALS) is a progressive, neurodegenerative disease in which motor neurons die. Since the ...
Does CRISPR Gene Editing Increase the Risk of Developing Cancer?
 CRISPR is a useful tool in a wide array of applications, and one of the most ...
