A new study presents a closed-loop gene therapy approach that targets only overactive neurons, reducing spontaneous seizures in mice. The approach uses the Fos gene to control the Kcna1 gene, promoting reduced neuronal excitability and offering a persistent antiepileptic effect.
Researchers at the University of Pennsylvania School of Medicine have developed a gene therapy that rapidly restores night vision in adults with LCA, a common form of congenital blindness. The treatment has been shown to improve rod-based visual functions and correct severe visual deficits in patients.
Researchers developed new educational materials to help individuals with sickle cell disease and their support networks learn about gene therapy clinical trials, benefits, risks, and treatment options. The project aims to facilitate engagement between the community and those conducting relevant clinical trials.
Researchers have developed a gene therapy that rescues cilia defects in retinal cells affected by Leber congenital amaurosis, a disease causing blindness in early childhood. The therapy restores opsin protein concentration in photoreceptor outer segments, suggesting functional NPHP5 may stabilize the primary cilium gate.
Researchers from Trinity College Dublin have developed a new gene therapy approach targeting mitochondrial function to treat dry AMD, a debilitating eye disease. The therapy has shown benefit in multiple models of the condition, offering hope for a potential treatment that could help millions globally.
A new type of adeno-associated virus (AAV) gene therapy candidate, FLT180a, has been shown to reduce bleeding risk in patients with haemophilia B. The treatment led to sustained production of FIX protein from the liver in nine out of ten patients, eliminating the need for regular replacement therapy.
A large cohort study found that hormone replacement therapy for breast cancer survivors is not associated with an increased risk of recurrence. The study included over 8,400 Danish postmenopausal women and found no link between vaginal estrogen or menopausal hormone therapy and breast cancer reoccurrence.
Researchers aim to develop a gene therapy that could suppress HIV replication and eliminate the need for life-long antiretroviral therapy. The study uses an AAV vector to produce leronlimab, a monoclonal antibody blocking HIV entry into immune cells.
The North American Menopause Society has released its 2022 Hormone Therapy Position Statement, reaffirming the effectiveness of hormone therapy in treating hot flashes and other menopause symptoms. The updated statement also highlights its role in preventing bone loss and fractures.
Researchers have developed a gene therapy that appears to correct a rare creatine deficiency disorder by increasing creatine levels and reducing toxic guanidinoacetic acid. This could hold promise for treating the disorder, which commonly results in intellectual disabilities and seizures.
A proof-of-concept study suggests that postnatal gene therapy may prevent or reverse deleterious effects of Pitt-Hopkins syndrome, a rare genetic disorder. The treatment restored normal activity of the deficient gene, preventing anxiety-like behavior and memory problems in an animal model.
A gene therapy trial has shown significant reduction in infections and improvement in eczema symptoms in children with Wiskott-Aldrich syndrome (WAS). The treatment, using lentiviral gene therapy, offers a safe alternative to bone marrow transplantation for patients without suitable donors.
Researchers at ChristianaCare's Gene Editing Institute describe a new process for evaluating the impacts of gene edits that alter rather than completely disabling DNA code. The study validates the safety and efficacy of their novel approach for using CRISPR to improve lung cancer treatments.
Researchers discovered a crucial RNA strand called CYTOR that helps build muscle mass, and found it decreases with age. Gene therapy stimulated CYTOR production, leading to increased fast-twitch muscle fibers and improved muscle function in humans and mice.
A recent study found that gene therapy delivery vectors were unlikely to cause the blood malignancies reported in trials; however, the exact cause remains a mystery. The study's results suggest that cancer risk in sickle cell disease may be more complex than initially thought, warranting further investigation.
Researchers found that dexamethasone administration after gene therapy increased liver transgene expression in mice. This finding suggests a potential strategy for reducing steroid side effects in future clinical trials.
A recent study led by University at Buffalo researchers found that photobiomodulation reduces inflammation, improves blood flow and heals wounds up to 19 days faster. Light therapy accelerated skin healing from cancer radiation therapy by an average of 49-42 days.
A novel gene therapy approach using an adeno-associated virus (AAV) has shown promising results in treating Huntington's disease. The therapy targets the mutated huntingtin gene (HTT), providing efficient and precise processing of a primary artificial microRNA, leading to potent pharmacological activity for HTT lowering.
Researchers developed a gene therapy called Targeted Augmentation of Nuclear Gene Output (TANGO), which boosts SCN1A protein production in brain cells. The treatment restored normal cell function and reduced seizures in lab mice with Dravet syndrome, offering hope for the first direct treatment of the fundamental cause.
A meta-analysis published in The Lancet Oncology reveals that men with intermediate- and high-risk prostate cancer experience improved overall survival with the addition of hormone therapy to radiotherapy. The study found prolonged adjuvant hormone therapy benefits men, while neoadjuvant hormone therapy did not.
Researchers developed a non-muscle targeted gene therapy that enhances muscle fiber repair and improves muscle function in LGMD 2B patients. The treatment, administered via a single injection, reduces muscle degeneration and restores myofiber size and muscle strength.
A novel gene delivery technique has successfully restored hearing in mice with a mutated STRC gene, which is the second most common genetic cause of hearing loss. The approach could be used to deliver other large genes and may provide a broad window of opportunity for treatment from babies to adults.
A new gene therapy called LentiGlobin has been shown to completely eliminate episodes of severe pain caused by sickle cell disease and restore blood cells to their normal shape. The treatment uses a patient's own stem cells and has the potential to give people with this disease their life back.
Researchers have developed a CRISPR/Cas9 gene editing system to enhance the effectiveness of sonodynamic therapy, allowing tumors to be effectively shrunk in a mouse model of liver cancer. The technology reduces antioxidant defense systems, increasing cancer cell death from the treatment.
A Charité study confirms the efficacy of gene therapy in improving muscle function and reducing mortality in children with spinal muscular atrophy. The treatment, onasemnogene abeparvoven, was found to be effective in children under two years of age, offering a promising alternative to existing treatments.
A novel gene therapy has shown sustained expression of clotting factor VIII, leading to a reduction or complete elimination of bleeding events in patients with hemophilia A. The trial demonstrated improved production of coagulation factor VIII over prolonged periods.
Researchers from Genethon and Université de Paris have developed an AAV-based gene therapy that corrects skeletal damage caused by XLH in mouse models. The treatment targets liver hepatocytes to express therapeutic proteins, potentially offering a lifelong solution for this rare disease.
A recent study by Sanford Burnham Prebys and the National Cancer Institute has shown that CRISPR-Cas9 gene editing can favor cells with mutated forms of genes linked to cancer. This highlights the importance of monitoring patients undergoing CRISPR-based gene therapy for cancer-related mutations.
A preclinical study shows that gene therapy can restore dopamine production in the brain, eliminating symptoms of Parkinson's disease. The therapy targets the substantia nigra region, where dopamine-releasing neurons reside, and provides a potential target for disease-modifying therapies.
A new study finds that high-dose radiation therapy can lengthen progression-free survival for people with advanced lung cancer when systemic therapy has not fully halted tumor growth or spread. Stereotactic body radiation therapy (SBRT) shows promise in treating oligoprogressive, metastatic lung and breast cancer.
Researchers report that 90% of patients who received gene therapy between 2009 and 2012 remain disease-free, with significant immune system differences observed among those treated. The therapy is most effective in younger children, but further work is needed to achieve high levels of gene correction in all patients.
A new study finds that left-sided breast cancer radiation therapy increases the risk of coronary artery disease in younger survivors, with a significant increase in risk seen at 10.5% for those who received left-sided radiation therapy compared to 5.8% for right-sided.
Researchers at Universitat Autonoma de Barcelona develop gene therapy that fully corrects severe whole-body alterations in Morquio A disease, a rare condition caused by GALNS enzyme deficiency. The therapy, administered via intravenous viral vector, prevents bone malformations and life-threatening complications.
Researchers developed a 'frameshifting' therapy that alters mRNA to reduce mast cell tumor size and prevent organ infiltration. The treatment, targeting the c-KIT gene, shows promise in reducing KIT protein expression, signaling, and function.
The Massage Therapy Foundation will host the International Massage Therapy Research Conference (IMTRC) on May 12-13, 2022, in Alexandria, Va. The event aims to bring together global experts to share research findings and discuss current innovations in massage therapy.
Researchers at Children's Hospital of Philadelphia have developed a system that can fine-tune protein expression from gene therapy vectors, addressing the need for controlled dosing. The 'dimmer switch' uses alternative RNA splicing and an orally available small molecule to adjust levels of expression up or down as needed.
A new study suggests that gene therapy delivered into the brain may be safe and effective in treating AADC deficiency. The therapy resulted in clinical improvement of symptoms, including reduced oculogyric crises, improved movement and motor function, and better sleep patterns.
A new study reveals that light therapy's benefits come from activating the Period1 gene in a brain region involved in mood regulation. Exposure to light at the end of the dark period has an antidepressant effect on mice.
Researchers identified a new gene, TP73, associated with an increased risk of sporadic ALS. Mutations in this gene have been found to interfere with nerve cell health and lead to abnormal cell differentiation and increased cell death. This discovery provides a potential target for therapy development.
Researchers developed a novel gene therapy approach that utilizes acetaminophen to correct disease-causing mutations and make liver cells immune to its toxic effects. This allows treated cells to multiply and reduces the need for liver transplants in treating genetic disorders.
Researchers have developed a platform that produces bespoke star-shaped polypeptide nanoparticles, effectively delivering range of therapies including gene therapies. The material accelerates bone tissue regeneration with a six-fold increase in new bone formation.
Research suggests that Nintendo Wii therapy can significantly improve balance in children with cerebral palsy, even when combined with conventional physical therapy. The analysis found medium to large effects of the therapy on functional balance and dynamic balance, respectively.
Researchers have found that a truncated version of the otoferlin gene, including its transmembrane domain, is essential for proper neurotransmitter release and sensory cell maturation. This discovery brings scientists closer to developing gene therapy treatments for severe congenital hearing loss.
A new form of gene therapy has successfully treated 48 out of 50 children born with a rare and deadly inherited disorder. The treatment, developed by an international team, involves delivering a corrected copy of the ADA gene into stem cells, which are then returned to the child's body to produce healthy immune cells.
Researchers developed a gene therapy that successfully treated 48 out of 50 children with ADA-SCID, a rare life-threatening disease. The treatment involves removing stem cells, delivering a new copy of the ADA gene, and returning the corrected cells to produce healthy immune cells.
Infants and children with ADA-SCID, a life-threatening inherited immunodeficiency disorder, have regained immune system function after receiving gene therapy. The treatment involves inserting a normal copy of the ADA gene into blood-forming stem cells, offering a one-time procedure with long-term benefits.
Scientists at the University of Colorado Anschutz Medical Campus discovered that intense light can enhance the expression of the ANGPTL4 gene, which regulates fatty lipids in plasma. This could lead to a new tool for safely blocking the gene and reducing cardiovascular risk.
Nanoscope Technologies is featuring groundbreaking research on optical gene delivery and functional characterization platform technologies for treating retinal diseases. The company's lead product, vMCO-010, delivers light-sensitive Multi-Characteristics Opsin into retinal cells to restore vision in patients with degeneration.
A new study by UCLA researchers identified a novel combination therapy using immune checkpoint inhibitors with ATRA to potentially overcome resistance to immunotherapy in LKB1-deficient lung cancer. The therapy led to eradication of over 70% of tumors and generated durable tumor-specific immunity.
CRISPR/Cas technology has advanced diagnostics and gene therapy, enabling fast and accurate disease diagnosis and treating untreatable diseases like cancer and blood disorders. Recent advances in CRISPR-Cas technologies are highlighted, with a discussion of the strength and challenges for its future clinical usage.
Researchers developed a new approach called Co-opting Regulation Bypass Repair (CRBR) that can correct genetic diseases caused by single gene mutations. The method uses the CRISPR/Cas9 system and non-homologous end joining to insert a functional copy of the gene, enabling treatment in all adult tissues.
The Tomographer algorithm transforms gene-sequencing data into spatially resolved images, mapping gene expression patterns across tissues. By analyzing tissue strips and mRNA measurements, the algorithm reconstructs spatial gene-expression patterns in tissues like the brain of the Australian Bearded Dragon.
Researchers at Penn Medicine delivered a new gene therapy that improved vision in three patients with severe vision impairments. The therapy showed sustained improvements in day and night vision, without serious side effects.
The Alliance for Cancer Gene Therapy Summit 2021 features key findings on translating CAR T-cell therapy success to complex solid tumors. Researchers share insights on evolving cell and gene regulatory processes, innovative science from the lab to the clinic, and tackling challenges in pancreatic cancer and glioblastoma.
A gene therapy protects eye cells in mice with a rare disorder, suggesting a combination approach may preserve vision in people with retinitis pigmentosa. Researchers found that using Txnip gene therapies, along with treatments for oxidative stress and inflammation, provided additional protection for the cells.
Xue Sherry Gao, a bioengineer at Rice University, has won an NIH grant to develop tools that can correct multiple single mutations causing cystic fibrosis. Her approach uses CRISPR/Cas9-based gene editing to fix the faulty CFTR gene, which is difficult to treat with traditional gene therapy.
A peptide nucleic acid-based gene editing technique has been identified as a crucial part of the gene editing toolkit, offering an alternative to CRISPR-Cas systems. The technique, developed by Carnegie Mellon's Danith Ly and Yale University's Peter Glazer, allows for in vivo gene editing using nanoparticles.
Scientists at the University of Cambridge have demonstrated a new approach to gene therapy that may help repair damage caused by neurodegenerative conditions like glaucoma and dementia. The study, published in Science Advances, shows promise for treating chronic degenerative diseases.
A study found that gene therapy can create a long-term store of correct T cells in the human thymus, leading to sustained health in patients with SCID-X1. The treatment works by delivering corrected genes into stem cells, which then produce healthy immune cells.
Researchers at UC San Diego developed a gene therapy that temporarily represses a gene involved in sensing pain, increasing pain tolerance and providing months of relief. The therapy could be used for various chronic pain conditions, including lower back pain and rare neuropathic disorders.