Voyager Therapeutics entered a strategic collaboration and capsid license agreement with Novartis to advance potential gene therapies for Huntington’s disease and spinal muscular atrophy, providing Novartis a target-exclusive license to access its TRACER capsids and other intellectual property.
Novartis obtains target-exclusive access to Voyager’s TRACER capsids related to Huntington’s disease and spinal muscular atrophy.
The world’s first partial heart transplant has achieved what researchers have spent more than a year hoping for—functioning valves and arteries that grow along with the young patient, as hypothesized by the pioneering team behind the procedure at Duke Health.
The procedure was performed in the spring of 2022, in an infant who needed heart valve replacement. The previous standard of care—using valves that were non-living—would not grow along with the child, requiring frequent replacement, entailing surgical procedures that carry a 50% mortality rate.
A study led by Duke Health physicians, appearing online Jan. 2 in the Journal of the American Medical Association (JAMA), found that the new manner of valve procurement used during the partial heart transplant led to two well-functioning valves and arteries that are growing in concert with the child as if they were native vessels.
2024 is expected to be the year when drone delivery finally takes flight.
What’s different about this year?
Well, most regulatory hurdles have been cleared, opening the door for retailers, medical centers, and logistics platforms to start offering drone delivery.
During testing, visual spotters were required every mile. Last Fall, the FAA authorized some drone operators to fly BVLOS (beyond visual line of sight). Now, companies such as Zipline, Wing, DroneUp, and Amazon are about to take off.
Just like a doctor adjusts the dose of a medication to the patient’s needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also needs to be maintained within a therapeutic window. Staying within the therapeutic window is important as too much of the protein could be toxic, and too little could result in a small or no therapeutic effect.
Although the principle of therapeutic window has been known for a long time, there has been no strategy to implement it safely, limiting the potential applications of gene therapy in the clinic.
In their current study published in the journal Nature Biotechnology, researchers at Baylor College of Medicine report on a technology to effectively regulate gene expression, a promising solution to fill this gap in gene therapy clinical applications. A Research Briefing on the breakthrough has been published in the same journal issue.
Although artificial skins can facilitate the healing of damaged skin, the restoration of tactile functions remain a challenge. Here, Kang et al. report an artificial skin with an implantable tactile sensor that can simultaneously replace the tactile function by nerve stimulation and promote skin regeneration.
Unlike older methods that use things foreign to our bodies, this one doesn’t trigger our immune system and employs small molecules to interact with RNA.
Researchers at Baylor College of Medicine have developed a breakthrough technology to regulate gene expression in gene therapy, addressing the crucial issue of maintaining therapeutic gene levels within a safe range.
This is important because having too much or too little of a gene’s activity within a therapeutic window can cause problems. Their method uses tiny substances in amounts approved by the FDA to control the genes.
Current methods have issues
The current methods for regulating genes have some problems, like causing harmful immune responses.
Diets that are higher in fat and significantly lower in carbohydrates are known to have a drastic effect on reducing the incidence of seizures in individuals with drug-resistant forms of epilepsy, particularly among children.
While it’s becoming apparent the diet creates some sort of shift in the gut’s microflora, the precise nature of those changes and their connection to the prevalence of seizures remains a mystery.
In a prospective study on children and experiments involving mice, researchers from the University of California, Los Angeles (UCLA) bring us a step closer to understanding how the foods we eat alter the functions of microbes in our digestive system, which in turn affect a variety of neurological functions suspected to play a role in epilepsy.
Just as healthy organs are vital to our well-being, healthy organelles are vital to the proper functioning of the cell. These subcellular structures carry out specific jobs within the cell; for example, mitochondria power the cell, and lysosomes keep the cell tidy.
Although damage to these two organelles has been linked to aging, cellular senescence, and many diseases, the regulation and maintenance of these organelles have remained poorly understood. Now, researchers at Osaka University have identified a protein, HKDC1, that plays a key role in maintaining these two organelles, thereby acting to prevent cellular aging.
There was evidence that a protein called TFEB is involved in maintaining the function of both organelles, but no targets of this protein were known. By comparing all the genes of the cell that are active under particular conditions and by using a method called chromatin immunoprecipitation, which can identify the DNA targets of proteins, the team was the first to show that the gene encoding HKDC1 is a direct target of TFEB, and that HKDC1 becomes upregulated under conditions of mitochondrial or lysosomal stress.
Memorial Sloan Kettering Cancer Center (MSK) has spearheaded revolutionary research unveiling groundbreaking strides in cancer treatment and understanding disease mechanisms.
Their discoveries include CAR T cell therapy targeting specific antigens in acute myeloid leukemia (AML), insights into the genetic element LINE-1, revelations on blood stem cell regulation, and a promising immunotherapy technique targeting CD47, showcasing potential breakthroughs in cancer therapy.
In the battle against acute myeloid leukemia (AML), traditional CAR T cell therapies faced hurdles due to varying antigens in AML cells and their similarity to normal blood stem cells, risking broader immune system damage.