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 ...
Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease
It is always exciting to observe phenomenal results in the treatment of a disease in animal studies, and even more so to see them move into clinical trials. ASOs have proven to be efficient at providing deep reductions in disease causing proteins, and a treatment that produced significant results in rats is now entering a clinical trial to treat ...
Can an ASO Reduce Disease Progression in ALS?
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 to ...
IKARIA – An Innovative Platform that Yields Long-Acting siRNA
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 ...
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 ...
Clinical Trials Begin for Ultra-Rare Treatment of Alexander Disease
It is always exciting to observe phenomenal results in the treatment of a disease in ...
Can an ASO Reduce Disease Progression in ALS?
What if you noticed your body acting a bit “off,” doing something unusual and more than ...
IKARIA – An Innovative Platform that Yields Long-Acting siRNA
In this year that marks the 20th Anniversary of the discovery of RNA interference as ...
