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Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.
The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.
For years, therapy dogs have ruled the world of animal-assisted services (AAS), offering stress relief to college students, hospital patients, and those in need of emotional support.
Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. But a major hurdle has been finding a safe and efficient way to deliver those genes.
A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.
This study marks an important step toward regenerative therapies to prevent heart failure. In mice, this protein was able to restore the heart by activating dormant repair genes without causing side effects, such as heart enlargement.
Researchers conducted the first clinical trial of an AI-powered therapy chatbot and found that, on average, people with diagnosed mental disorders experienced clinically significant improvements in their symptoms over eight weeks, according to new results.
In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.
Regenerative heart therapies involve transplanting cardiac muscle cells into damaged areas of the heart to recover lost function. However, the risk of arrhythmias following this procedure is reportedly high.
Treatment typically involves surgery and follow-up hormone therapy, but late effects of these treatments include osteoporosis, sexual dysfunction and blood clots. Despite significant therapeutic advances, breast cancer remains a leading cause of cancer-related death in women.
A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source localization, ability to hear in noisy environments, and for two children, abillity to appreciate music.
Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an international Phase III clinical trial.
An experimental Alzheimer’s therapy from Cassava Sciences is still being tested in two Phase 3 studies, even as the company has come under regulatory scrutiny.
One day, researchers want to use switches of this kind to trigger cell therapies for various metabolic diseases. Researchers have developed a new gene switch that can be activated using a commercially available nitroglycerine patch applied to the skin.
In the rapidly advancing field of cell therapies, Dr Jason Bock has emerged as a leader, known for his innovative approach to optimising the development process. With over 25 years of experience in therapeutics, Bock has played a pivotal role in shaping the future of cell therapies, particularly through his work at CTMC.
In April, the FDA warned of risk of secondary cancers in people receiving CAR-T cell therapy. A large study now finds the risk is low and not related to the CAR-T cells.
Researchers have found that two of the most frequently administered stem cell therapies, which are often used interchangeably, actually contain completely different types of cells.
The mucus in the airways is not as sticky, inflammation in the lungs significantly reduced: Triple combination therapy can achieve these positive, lasting effects in patients with cystic fibrosis (CF). According to new research, this form of medication improves the symptoms of CF in many patients.
A novel gene therapy for hearing loss was administered to six children in China in a clinical trial. Each child had an inherited deafness caused by mutations in the OTOF gene, called DFNB9.
A broad new strategy could hold hope for treating virtually all blood cancers with CAR T cell therapy, which is currently approved for five subtypes of blood cancer. Scientists have demonstrated the potential efficacy of this approach a preclinical study.
Researchers devised a process to convert a skin cell directly into a neuron, eliminating the need to generate induced pluripotent stem cells. Such neurons could be used to treat spinal cord injuries or diseases such as ALS.
They also found that co-transplantation of neuronal cell therapy with host regulatory T cells resulted in effective suppression of needle trauma and significant improvement in the survival and recovery of grafts. These findings suggest a path for the 'realistic' use of cell therapy to treat neurodegenerative disorders.
Scientists have found that statins -- commonly used cholesterol-lowering drugs -- may block a pathway that leads to the development of cancer in the context of chronic inflammation.
Results demonstrated the safety and preliminary efficacy of the novel gene therapy approach in high-grade glioma patients, with prolonged survival in a subgroup of recurrent GBM patients immunologically 'familiar' with the virus.
Elsewhere, AskBio advanced a gene therapy and Pfizer hired a scientist well versed in GLP-1 science. The Swiss pharma will base cardiometabolic drug research at the new Allston site.
With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.
Scientists have made a `paradigm shifting' discovery on the mechanisms required for learning and memory that could lead to new therapies for Alzheimer's disease and potentially Down syndrome.
This discovery opens up new opportunities for therapeutic intervention to control cholesterol uptake that could complement other therapies and potentially save lives. Researchers have discovered the mechanism by which cholesterol in our diet is absorbed into our cells.
Menthol sensing appeared long before cold sensing, suggesting distinct activation modes that can be disentangled, paving the way for new pain therapies without adverse thermal side effects.
A large-scale clinical trial of treatment strategies for Crohn's disease has shown that offering early advanced therapy to all patients straight after diagnosis can drastically improve outcomes, including by reducing the number of people requiring urgent abdominal surgery for treatment of their disease by ten-fold.
Promising preclinical results show hematopoietic stem cell therapy was effective in rescuing memory loss, neuroinflammation and beta amyloid build-up in a mouse model of Alzheimer's disease.
Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach. Meet Mark Bagnall Mark Bagnall currently serves as CEO of Phenomix Sciences, Inc.
Pain experts suggest that scrambler therapy, a noninvasive pain treatment, can yield significant relief for approximately 80% -- 90% of patients with chronic pain, and it may be more effective than another noninvasive therapy: transcutaneous electrical nerve stimulation (TENS).
Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If
Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain.
An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells. Lisa Yang and Hock E.
Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.
A 'loopy' discovery in bacteria is raising fundamental questions about the makeup of our own genome -- and revealing a potential wellspring of material for new genetic therapies.
• Face-to-face event, taking place in London, UK, 26-27th April, will feature two dedicated tracks: ‘Scientific’, focused on seven core therapy areas and ‘Life Sciences Business’ Programme features keynote from George Freeman MP, with an update on the UK Government Life Sciences VisionRegistration for BioTrinity 2022 now available online
New therapy uses synthetic nanofibers to mimic the natural signaling of a protein that is crucial for cartilage formation and maintenance. Therapy could be used to treat osteoarthritis, which affects nearly 530 million people worldwide. After just four hours, the treatment activated the gene expression necessary to generate cartilage.
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